FARA - Site Map

Plasma and red blood cell membrane accretion and pharmacokinetics of RT001 (bis-allylic 11,11-D2-linoleic acid ethyl ester) during long term dosing in patients

Friedreich Ataxia: current state-of-the-art, and future prospects for mitochondrial-focused therapies

Inherited Cerebellar Ataxias: 5-Year Experience of the Irish National Ataxia Clinic

Atypical structures of GAA/TTC trinucleotide repeats underlying Friedreich's ataxia: DNA triplexes and RNA/DNA hybrids

Reata provides update on development of omaveloxolone in FA

R-loops promote trinucleotide repeat deletion through DNA base excision repair enzymatic activities

Onset features and time to diagnosis in Friedreich's Ataxia

A Drosophila model of Friedreich Ataxia with CRISPR/Cas9 insertion of GAA repeats in the frataxin gene reveals in vivo protection by N-acetyl cysteine

Antioxidant Defense Mechanisms and its Dysfunctional Regulation in the Mitochondrial Disease, Friedreich's Ataxia

Toward quantitative neuroimaging biomarkers for Friedreich's ataxia at 7 Tesla: Susceptibility mapping, diffusion imaging, R 2 and R 1 relaxometry

Antioxidant Therapies and Oxidative Stress in Friedreich´s Ataxia: The Right Path or Just a Diversion?

Larimar Therapeutics Announces Dosing of Patients in Third Cohort of Phase 1 SAD Trial of CTI-1601 for Treatment of Friedreich’s Ataxia

Current Status of microRNA-Based Therapeutic Approaches in Neurodegenerative Disorders

Safety and Efficacy of (+)-Epicatechin in Subjects with Friedreich's Ataxia: A Phase II, Open-Label, Prospective Study

MR imaging and spectroscopy in degenerative ataxias: toward multimodal, multisite, multistage monitoring of neurodegeneration

Cardiolipin-deficient cells have decreased levels of the iron-sulfur biogenesis protein frataxin

Compound heterozygosity for an expanded (GAA) and a (GAAGGA) repeat at FXN locus: from a diagnostic pitfall to potential clues to the pathogenesis of Friedreich ataxia

Trinucleotide Repeat Disorders

Stress-induced Mouse Model of the Cardiac Manifestations of Friedreich's Ataxia Corrected by AAV-mediated Gene Therapy

A Novel Solution-Gated Graphene Transistor Biosensor for Ultrasensitive Detection of Trinucleotide Repeats

New Request For Proposals: Therapeutic window of frataxin

Feasibility and Acceptability of Lee Silverman Voice Treatment in Progressive Ataxias

Treatment of Dilated Cardiomyopathy in a Mouse Model of Friedreich's Ataxia Using N-acetylcysteine and Identification of Alterations in MicroRNA Expression That Could Be Involved in Its Pathogenesis

Defining Transcription Regulatory Elements in the Human Frataxin Gene: Implications for Gene Therapy

Vestibular Impact of Friedreich Ataxia in Early Onset Patients

Cerebellar Cognitive Disorder Parallels Cerebellar Motor Symptoms in Friedreich Ataxia

Significance of NT-proBNP and High-sensitivity Troponin in Friedreich Ataxia

Hereditary Ataxia: A Focus on Heme Metabolism and Fe-S Cluster Biogenesis

Chondrial Therapeutics and Zafgen Complete Merger and Begin Operating as Larimar Therapeutics

Making iron-sulfur cluster: structure, regulation and evolution of the bacterial ISC system

Oxidative Stress in DNA Repeat Expansion Disorders: A Focus on NRF2 Signaling Involvement

Neurologic Outcomes in Friedreich Ataxia: Study of a Single-Site Cohort

Therapeutic Potential of Stem Cells for Treatment of Neurodegenerative Diseases

ExpansionHunter Denovo: A Computational Method for Locating Known and Novel Repeat Expansions in Short-Read Sequencing Data

Oxidative Stress, a Crossroad Between Rare Diseases and Neurodegeneration

Outlining the Complex Pathway of Mammalian Fe-S Cluster Biogenesis

A Comprehensive Transcriptome Analysis Identifies FXN and BDNF as Novel Targets of miRNAs in Friedreich's Ataxia Patients

An Overview of the Current State and the Future of Ataxia Treatments

Calpain-Inhibitors Protect Frataxin-Deficient Dorsal Root Ganglia Neurons from Loss of Mitochondrial Na+/Ca2+ Exchanger, NCLX, and Apoptosis

Cofilin dysregulation alters actin turnover in frataxin-deficient neurons

Particle-mediated delivery of frataxin plasmid to a human sensory neuronal model of Friedreich's ataxia

Frataxin-deficient cardiomyocytes present an altered thiol-redox state which targets actin and pyruvate dehydrogenase

A wearable video-oculography based evaluation of saccades and respective clinical correlates in patients with early onset ataxia

Neurophysiologic intraoperative monitoring (NIOM) in pediatric patients with polyneuropathy

Identification of Frataxin as a regulator of ferroptosis

Inherited Metabolic Diseases and Cardiac Pathology in Adults: Diagnosis and Prevalence in a CardioMetabo Study

Management of Neuroinflammatory Responses to AAV-Mediated Gene Therapies for Neurodegenerative Diseases

On the wrong DNA track: Molecular mechanisms of repeat-mediated genome instability

The Potential of the Novel NAD+ Supplementing Agent, SNH6, as a Therapeutic Strategy for the Treatment of Friedreich's Ataxia

Ocular Involvement in Friedreich Ataxia Patients and its Relationship with Neurological Disability, a Follow-up Study

The NRF2 Signaling Network Defines Clinical Biomarkers and Therapeutic Opportunity in Friedreich's Ataxia

Mechanisms of Mitochondrial Iron-Sulfur Protein Biogenesis

Developing an objective evaluating system to quantify the degree of upper limb movement impairment in patients with severe Friedreich's ataxia

NfL and pNfH are increased in Friedreich's ataxia

Safety and Efficacy of Interferon γ in Friedreich's Ataxia

Frataxin deficiency in Friedreich's ataxia is associated with reduced levels of HAX-1, a regulator of cardiomyocyte death and survival

Large-scale contractions of Friedreich's ataxia GAA repeats in yeast occur during DNA replication due to their triplex-forming ability

MDA Awards Venture Philanthropy Funding of More Than $1M to AavantiBio to Develop Gene-Targeting Therapy for Friedreich's Ataxia

Mammalian iron-sulfur cluster biogenesis: Recent insights into the roles of frataxin, acyl carrier protein and ATPase-mediated transfer to recipient proteins.

Retrotope Expands its Drug Pipeline with the First Dosing of RT001 in patients with Friedreich’s ataxia (FA)

Genetic Analysis of Hereditary Ataxias in Peru Identifies SCA10 Families with Incomplete Penetrance

Nanoscopic X-ray imaging and quantification of the iron cellular architecture within single fibroblasts of Friedreich's ataxia patients

A new tool to determine the cellular metabolic landscape: nanotechnology to the study of Friedreich's ataxia

Neurochemical profiles in hereditary ataxias: A meta-analysis of Magnetic Resonance Spectroscopy studies

Iron Hack - A symposium/hackathon focused on porphyrias, Friedreich's ataxia, and other rare iron-related diseases

Zafgen and Chondrial Therapeutics Announce Definitive Merger Agreement

Effects of tocotrienol supplementation in Friedreich's ataxia: A model of oxidative stress pathology

Friedreich's Ataxia: Case series and the Additive Value of Cardiovascular Magnetic Resonance

Pediatric Neuromuscular Disorders

N-Terminomics/TAILS Profiling of Macrophages after Chemical Inhibition of Legumain

Possible Mechanisms of Biological Effects Observed in Living Systems during 2H/1H Isotope Fractionation and Deuterium Interactions with Other Biogenic Isotopes

Left ventricular structural and functional changes in Friedreich ataxia - Relationship with body size, sex, age and genetic severity

Global Ataxia Advocacy Organizations Unite to Host International Ataxia Research Conference in Washington, DC

Quantitative Proteomic and Network Analysis of Differentially Expressed Proteins in PBMC of Friedreich's Ataxia (FRDA) Patients

Refinement of protein Fe(II) binding characteristics utilizing a competition assay exploiting small molecule ferrous chelators

Evolutionarily conserved susceptibility of the mitochondrial respiratory chain to SDHI pesticides and its consequence on the impact of SDHIs on human cultured cells

Speech and Language Disorders in Friedreich Ataxia: Highlights on Phenomenology, Assessment, and Therapy

Potential biomarker identification for Friedreich's ataxia using overlapping gene expression patterns in patient cells and mouse dorsal root ganglion

The first biallelic missense mutation in the FXN gene in a consanguineous Turkish family with Charcot-Marie-Tooth-like phenotype

Targeting NRF2 for the Treatment of Friedreich's Ataxia: A Comparison among Drugs

Cerebellum and cognition in Friedreich ataxia: a voxel-based morphometry and volumetric MRI study

Predictors of Left Ventricular Dysfunction in Friedreich's Ataxia in a 16-Year Observational Study

Induced pluripotent stem cells-derived neurons from patients with Friedreich ataxia exhibit differential sensitivity to resveratrol and nicotinamide

Minoryx Therapeutics receives FDA Orphan Drug Designation for leriglitazone in Friedreich’s Ataxia

SINEUP non-coding RNAs rescue defective frataxin expression and activity in a cellular model of Friedreich's Ataxia

Minoryx Therapeutics completes enrollment in FRAMES phase 2 trial with leriglitazone in Friedreich's Ataxia

Health-related quality of life and depressive symptoms in Friedreich ataxia

Unique roles of iron and zinc binding to the yeast Fe-S cluster scaffold assembly protein "Isu1"

Mechanism of activation of the human cysteine desulfurase complex by frataxin

Two new Pfizer-coauthored studies validate Novoheart’s pioneering human bioengineered heart tissues and chambers for improving drug development

Scoliosis in Patients With Friedreich Ataxia: Results of a Consecutive Prospective Series

Friedreich ataxia- pathogenesis and implications for therapies

The Working Life of People with Degenerative Cerebellar Ataxia

Backbone resonance assignments and secondary structure of the apo-Drosophila melanogaster frataxin homolog (Dfh)

Application of Quantitative Motor Assessments in Friedreich Ataxia and Evaluation of Their Relation to Clinical Measures

Oral mobility reflects rate of progression in advanced Friedreich's ataxia

Nrf2 Induction Re-establishes a Proper Neuronal Differentiation Program in Friedreich's Ataxia Neural Stem Cells

Physiologically relevant reconstitution of iron-sulfur cluster biosynthesis uncovers persulfide-processing functions of ferredoxin-2 and frataxin

Low frataxin mRNA expression is associated with inflammation and oxidative stress in patients with type 2 diabetes

REGENXBIO Announces New License Agreement with Pfizer for the Treatment of Friedreich's Ataxia Using NAV® AAV9 Vector

Correlation between frataxin expression and contractility revealed by in vitro Friedreich's ataxia cardiac tissue models engineered from human pluripotent stem cells

Frequency and Genetic Profile of Compound Heterozygous Friedreich's Ataxia Patients-the Brazilian Experience

FARA Job Posting: Associate Director of Research

Deuterated Polyunsaturated Fatty Acids Reduce Oxidative Stress and Extend the Lifespan of C. elegans

Young Investigators: Call for Abstracts, 12th Annual FA Symposium

p53 Binds Preferentially to Non-B DNA Structures Formed by the Pyrimidine-Rich Strands of GAA·TTC Trinucleotide Repeats Associated with Friedreich's Ataxia

Erythropoietin and Friedreich Ataxia: Time for a Reappraisal?

Peripapillary retinal nerve fibre layer thickness in Friedreich's ataxia: a biomarker for trials?

New Insights into the Hepcidin-Ferroportin Axis and Iron Homeostasis in iPSC-Derived Cardiomyocytes from Friedreich's Ataxia Patient

Hypoxia Rescues Frataxin Loss by Restoring Iron Sulfur Cluster Biogenesis

Mechanism of frataxin "bypass" in human iron-sulfur cluster biosynthesis with implications for Friedreich's ataxia

StrideBio and Takeda Sign Collaboration and License Agreement to Advance Novel Gene Therapies for Neurological Diseases

Patient reported outcomes in Friedreich's Ataxia after withdrawal from Idebenone

Assessment of cell-free levels of iron and copper in patients with Friedreich's ataxia

Inherited Ataxia and Intrathecal Baclofen for the Treatment of Spasticity and Painful Spasms

The role of robotic gait training and tDCS in Friedrich ataxia rehabilitation: A case report

Minoryx Therapeutics receives approval from Spanish regulatory agency to initiate Phase 2 Study in Friedreich's Ataxia

Guidelines on the diagnosis and management of the progressive ataxias

In Vitro interaction between yeast frataxin and superoxide dismutases: Influence of mitochondrial metals

The role of mitochondrial labile iron in Friedreich's ataxia skin fibroblasts sensitivity to ultraviolet A

Pattern of Cerebellar Atrophy in Friedreich's Ataxia-Using the SUIT Template

Phosphodiesterase Inhibitors Revert Axonal Dystrophy in Friedreich's Ataxia Mouse Model

Heart disease in Friedreich's ataxia

Neurocrine Biosciences and Voyager Therapeutics Form Strategic Development and Commercialization Collaboration for Parkinson’s Disease and Friedreich’s Ataxia

OGTT is recommended for glucose homeostasis assessments in Friedreich ataxia

Structural and functional characterization of a frataxin from a thermophilic organism

Longitudinal evaluation of iron concentration and atrophy in the dentate nuclei in friedreich ataxia

Drug repositioning screening identifies etravirine as a potential therapeutic for friedreich's ataxia

Correction of half the cardiomyocytes fully rescue Friedreich Ataxia mitochondrial cardiomyopathy through cell-autonomous mechanisms

Large Interruptions of GAA Repeat Expansion Mutations in Friedreich Ataxia Are Very Rare

Depressive symptoms in Friedreich ataxia

FAST-1 antisense RNA epigenetically alters FXN expression

Rapid and Complete Reversal of Sensory Ataxia by Gene Therapy in a Novel Model of Friedreich Ataxia

Oligonucleotides Hold Promise as a Therapy for Friedreich's Ataxia

Voyager Therapeutics Announces Additional Data at the American Society of Gene and Cell Therapy 2018 Annual Meeting

Safety, pharmacodynamics, and potential benefit of omaveloxolone in Friedreich ataxia

Plasma Markers of Neurodegeneration Are Raised in Friedreich's Ataxia

Intraepidermal Nerve Fiber Density in Friedreich's Ataxia

Developmental and Neurodegenerative Damage in Friedreich Ataxia

Corneal Confocal Microscopy: Neurologic Disease Biomarker in Friedreich's Ataxia

Automated functional upper limb evaluation of patients with Friedreich ataxia using serious games rehabilitation exercises

Iron in Friedreich Ataxia: A Central Role in the Pathophysiology or an Epiphenomenon

Functional and Structural Brain Damage in Friedreich's Ataxia

Sudomotor dysfunction is frequent and correlates with disability in Friedreich ataxia

Increased Frataxin Expression Induced in Friedreich Ataxia Cells by Platinum TALE-VP64s or Platinum TALE-SunTag

Test-retest reliability of an instrumented electronic walkway system (GAITRite) for the measurement of spatio-temporal gait parameters in young patients with Friedreich's ataxia

Two funded post-doctoral positions are available in the laboratory of Hélène Puccio at the IGBMC in Strasbourg.

Nonataxia symptoms in Friedreich Ataxia: Report from the Registry of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS)

RNA-Dependent Epigenetic Silencing Directs Transcriptional Downregulation Caused by Intronic Repeat Expansions

Zinc(II) binding on human wild-type ISCU and Met140 variants modulates NFS1 desulfurase activity

PTC Therapeutics to acquire Agilis Biotherapeutics

Young Investigators, Call for Abstracts for the 11th Annual Friedreich's Ataxia Symposium

Impact of Drosophila Models in the Study and Treatment of Friedreich's Ataxia

Autonomic function testing in Friedreich's ataxia

Structural characteristics of the central nervous system in Friedreich ataxia: an in vivo spinal cord and brain MRI study

Cognitive and functional connectivity alterations in Friedreich's ataxia

Adding a temporal dimension to the study of Friedreich's ataxia: the effect of frataxin overexpression in a human cell model

The importance of central auditory evaluation in Friedreich's ataxia

Diabetes mellitus in Friedreich Ataxia: A case series of 19 patients from the German-Austrian diabetes mellitus registry

Randomized, clinical trial of RT001: Early signals of efficacy in Friedreich's ataxia

Rapid exhaustion of auditory neural conduction in a prototypical mitochondrial disease, Friedreich ataxia

Iron regulatory protein deficiency compromises mitochondrial function in murine embryonic fibroblasts

Mitofusin-Dependent ER Stress Triggers Glial Dysfunction and Nervous System Degeneration in a Drosophila Model of Friedreich's Ataxia

Pitfalls in molecular diagnosis of Friedreich ataxia

Bone marrow transplantation stimulates neural repair in Friedreich's ataxia mice

Biophysical characterisation of the recombinant human frataxin precursor

Small RNA-seq analysis of circulating miRNAs to identify phenotypic variability in Friedreich's ataxia patients

RNA-DNA hybrids promote the expansion of Friedreich's ataxia (GAA)n repeats via break-induced replication

Serum uric acid in Friedreich Ataxia

Join Reata WebEx. Get updates on the MOXIe Phase 2 Study

Quantitative proteomics in Friedreich's ataxia B-lymphocytes: A valuable approach to decipher the biochemical events responsible for pathogenesis

The role of Nrf2 signaling in counteracting neurodegenerative diseases

Effect Of Diazoxide on Friedreich Ataxia Models

Emotion Recognition and Psychological Comorbidity in Friedreich's Ataxia

Friedreich and dominant ataxias: quantitative differences in cerebellar dysfunction measurements

Mitochondrial pore opening and loss of Ca2+ exchanger NCLX levels occur after frataxin depletion

The role of oxidative stress in Friedreich's ataxia

Synthetic transcription elongation factors license transcription across repressive chromatin

Insights on the conformational dynamics of human frataxin through modifications of loop-1

Peripheral Nerve Ultrasound in Friedreich's Ataxia

Personality and Neuropsychological Profiles in Friedreich Ataxia

Agilis Biotherapeutics Announces Orphan Product Designation Approval in Europe for the Treatment of Friedreich Ataxia

Transplantation of wild-type mouse hematopoietic stem and progenitor cells ameliorates deficits in a mouse model of Friedreich's ataxia

Friedreich's ataxia: clinical features, pathogenesis and management

Nrf2-Inducers Counteract Neurodegeneration in Frataxin-Silenced Motor Neurons: Disclosing New Therapeutic Targets for Friedreich's Ataxia

Reata Announces First Patient Enrolled in Part 2 of MOXIe Study of Omaveloxolone for the Treatment of Friedreich’s Ataxia

Friedreich Ataxia: Developmental Failure of the Dorsal Root Entry Zone

Sustained FXN expression in dorsal root ganglia from a nonreplicative genomic HSV-1 vector

BioMarin Highlights Breadth of Innovative Development Pipeline at R&D Day on Oct 18th in New York

The Subclinical Cardiomyopathy of Friedreich's Ataxia in a Pediatric Population

FDA awards six grants for natural history studies in rare diseases

Frataxin-deficient neurons and mice models of Friedreich ataxia are improved by TAT-MTScs-FXN treatment

Urinary, bowel and sexual symptoms in a cohort of patients with Friedreich's ataxia

Agilis Biotherapeutics Updates on Progress in CNS Gene Therapy Programs

Molecular Alterations in a Mouse Cardiac Model of Friedreich's Ataxia: An Impaired Nrf2 Response Mediated via Up-Regulation of Keap1 and Activation of the Gsk3β Axis

Reversible Axonal Dystrophy by Calcium Modulation in Frataxin-Deficient Sensory Neurons of YG8R Mice

LNA effects on DNA binding and conformation: from single strand to duplex and triplex structures

Defining the Architecture of the Core Machinery for the Assembly of Fe-S Clusters in Human Mitochondria

Peptide SS-31 upregulates frataxin expression and improves the quality of mitochondria: implications in the treatment of Friedreich ataxia

Overexpression of Drosophila frataxin triggers cell death in an iron-dependent manner

Part 2 of the Phase II MOXIe study (RTA 408 or omaveloxolone)

Cerebral Abnormalities in Friedreich Ataxia: A Review

Jupiter Orphan Therapeutics Receives Orphan Drug Designation for its Trans-Resveratrol Product JOTROL for Treatment of Friedreich's Ataxia

FDA Confirms That Use of mFARS as Primary Endpoint in Part 2 of the MOXIe Trial Can Support Approval of Omaveloxolone in Friedreich’s Ataxia

Mitochondrial dysfunction in the neuro-degenerative and cardio-degenerative disease, Friedreich's ataxia

Chondrial Announces FDA Orphan Drug Designation for CTI-1601, a Novel Investigational Technology for the Treatment of Friedreich's Ataxia

Call for Abstracts - 10th Annual Friedreich’s Ataxia Symposium

Circulating miR-323-3p is a biomarker for cardiomyopathy and an indicator of phenotypic variability in Friedreich's ataxia patients

Cerebral compensation during motor function in Friedreich ataxia: The IMAGE-FRDA study

Structural signature of classical versus late-onset friedreich's ataxia by Multimodality brain MRI

Erythropoietin and small molecule agonists of the tissue-protective erythropoietin receptor increase FXN expression in neuronal cells in vitro and in Fxn-deficient KIKO mice in vivo

Dimethyl Fumarate Mediates Nrf2-dependent Mitochondrial Biogenesis in Mice and Humans

Normalization of timed neuropsychological tests with the PATA rate and nine-hole pegboard tests

Dysphagia in Friedreich Ataxia

Mesenchymal Stem Cell-Derived Factors Restore Function to Human Frataxin-Deficient Cells

Frataxin Deficiency Impairs Mitochondrial Biogenesis in Cells, Mice and Humans

No changes in heme synthesis in human Friedreich´s ataxia erythroid progenitor cells

FARA Job Posting: Research Director

Call For Abstracts - 2nd International Ataxia Research Conference

Visiting FARA Funded Researchers

Rare Disease Day 2017 – Friedreich ataxia: Researchers from Fratagene Therapeutics and the University of Rome “Tor Vergata” identify a new therapeutic target

Call For Abstracts - 2nd International Ataxia Research Conference

E3 Ligase RNF126 Directly Ubiquitinates Frataxin, Promoting Its Degradation: Identification of a Potential Therapeutic Target for Friedreich Ataxia

Chondrial Therapeutics Secures Up to $22.6 Million in Series A Financing and Licenses Novel Technology for the Treatment of Friedreich’s Ataxia

Newly Funded FARA Grants

STEADFAST Background for the Friedreich's Ataxia community

Cytokine therapy-mediated neuroprotection in a Friedreich's ataxia mouse model

Deletion of the GAA repeats from the human frataxin gene using the CRISPR-Cas9 system in YG8R-derived cells and mouse models of Friedreich Ataxia

Mitochondrial capacity, muscle endurance & low energy in friedreich ataxia

Liver Growth Factor (LGF) Upregulates Frataxin Protein Expression and Reduces Oxidative Stress in Friedreich's Ataxia Transgenic Mice

Horizon Pharma plc Announces Topline Results from Phase 3 Study of ACTIMMUNE® (interferon gamma-1b) in Friedreich's Ataxia

Disruption of Higher Order DNA Structures in Friedreich's Ataxia (GAA)n Repeats by PNA or LNA Targeting

Save The Date - 2017 International Ataxia Research Conference

Heterologous mitochondrial targeting sequences can deliver functional proteins into mitochondria

Cognition in Friedreich's ataxia: a behavioral and multimodal imaging study

Functional and Gait Assessment in Children and Adolescents Affected by Friedreich's Ataxia: A One-Year Longitudinal Study

Pharmacological treatments for Friedreich ataxia

Neurobehavioral deficits in the KIKO mouse model of Friedreich's ataxia

Serum versus Imaging Biomarkers in Friedreich Ataxia to Indicate Left Ventricular Remodeling and Outcomes

Frataxin silencing alters microtubule stability in motor neurons: implications for Friedreich's Ataxia

Architecture of the Human Mitochondrial Iron-Sulfur Cluster Assembly Machinery

Cerebral and cerebellar grey matter atrophy in Friedreich ataxia: the IMAGE-FRDA study

Voice in Friedreich Ataxia

Long-term treatment with thiamine as possible medical therapy for Friedreich ataxia

Agilis Biotherapeutics Announces FDA Orphan Drug Designation for the Treatment of Friedreich’s Ataxia (FA)

Pfizer aims to become industry leader in gene therapy with acquisition of Bamboo Therapeutics, Inc.

9th Annual Friedreich’s Ataxia Symposium – 2016 CHOP/FARA – Call For Abstracts

The Replication of Frataxin Gene Is Assured by Activation of Dormant Origins in the Presence of a GAA-Repeat Expansion

Metal Homeostasis Regulators Suppress FRDA Phenotypes in a Drosophila Model of the Disease

Interferon gamma may improve cardiac function in Friedreich's ataxia cardiomyopathy

Crystal Structure of Bacillus subtilis Cysteine Desulfurase SufS and Its Dynamic Interaction with Frataxin and Scaffold Protein SufU

Downregulation of GSTK1 Is a Common Mechanism Underlying Hypertrophic Cardiomyopathy

Vestibulo-ocular reflex dynamics with head-impulses discriminates spinocerebellar ataxias types 1, 2 and 3 and Friedreich ataxia

Oxidative Stress and altered lipid metabolism in Friedreich ataxia

US FDA Grants Orphan Drug Designation for Retrotope’s RT001 in the treatment of Friedreich’s ataxia

Reata Pharmaceuticals, Inc. Announces Pricing of Initial Public Offering

Purkinje cell injury, structural plasticity and fusion in patients with Friedreich's ataxia

Plasma circulating cell-free mitochondrial DNA in the assessment of Friedreich's ataxia

Horizon Pharma plc to Acquire Worldwide Rights to Interferon Gamma-1b From Boehringer Ingelheim International GmbH

Meeting Notice: FARA's Biomarker Meeting, Sept 16, 2016

Job Opportunity: Postdoctoral Fellow Position open at University of Alabama at Birmingham

Horizon Pharma plc Completes Target Enrollment of 90 Patients for Phase 3 Trial of ACTIMMUNE(R) (interferon gamma-1b) for the Treatment of People With Friedreich's Ataxia

Frataxin and the molecular mechanism of mitochondrial iron-loading in Friedreich's ataxia

Time-resolved functional analysis of acute impairment of frataxin expression in an inducible cell model of Friedreich ataxia

Two different pathogenic mechanisms, dying-back axonal neuropathy and pancreatic senescence, are present in the YG8R mouse model of Friedreich ataxia

Development of an AAV9 coding for a 3XFLAG-TALEfrat#8-VP64 able to increase in vivo the human frataxin in YG8R mice

Lymphoblast Oxidative Stress Genes as Potential Biomarkers of Disease Severity and Drug Effect in Friedreich's Ataxia

The Structure of the Complex Between Yeast Frataxin and Ferrochelatase: Characterization and pre-Steady StateReaction of Ferrous Iron Delivery and Heme Synthesis

Clinical Experience With Deferiprone Treatment for Friedreich Ataxia

Iron chelation in the treatment of neurodegenerative diseases

Tissue atrophy and elevated iron concentration in the extrapyramidal motor system in Friedreich ataxia: the IMAGE-FRDA study

Frataxin Deficiency Promotes Excess Microglial DNA Damage and Inflammation that Is Rescued by PJ34

Architecture of the Yeast Mitochondrial Iron-Sulfur Cluster Assembly Machinery: The Sub-Complex Formed by the Iron Donor, Yfh1, and the Scaffold, Isu1

Reversal of epigenetic promoter silencing in Friedreich ataxia by a class I histone deacetylase inhibitor

Combined Cerebellar Proton MR Spectroscopy and DWI Study of Patients with Friedreich's Ataxia

Intrathecal delivery of frataxin mRNA encapsulated in lipid nanoparticles to dorsal root ganglia as a potential therapeutic for Friedreich's ataxia

Retrotope advances RT001 in clinical trials to treat Friedreich’s ataxia

Avalanche Biotechnologies and Annapurna Therapeutics announce proposed merger

Emerging therapies in Friedreich's ataxia

Nonneurological Involvement in Late-Onset Friedreich Ataxia (LOFA): Exploring the Phenotypes

Update on the STEADFAST study

Chondrial Therapeutics Announces Development Milestone

Jupiter Orphan Therapeutics, Inc. Enters into a Global Licensing Agreement with Murdoch Childrens Research Institute

FARA Grant Program Update

Annapurna Therapeutics to Collaborate with Weill Cornell Medicine on Gene-Therapy Portfolio

Longitudinal magnetic resonance imaging study shows progressive pyramidal and callosal damage in Friedreich's ataxia

Perturbation of cellular proteostasis networks identifies pathways that modulate precursor and intermediate but not mature levels of frataxin

Analyzing the Effects of a G137V Mutation in the FXN Gene

GIFT-1, a phase IIa clinical trial to test the safety and efficacy of IFNγ administration in FRDA patients

Efficient attenuation of Friedreich's ataxia (FRDA) cardiomyopathy by modulation of iron homeostasis-human induced pluripotent stem cell (hiPSC) as a drug screening platform for FRDA

Retrotope RT0001 Clinical Trial Update

Reata RTA 408 Clinical Trial Update

Retrotope announces opening of second clinical trial site for enrollment in Friedreich’s ataxia clinical trial.

Funding Announcement: Clinical Trial Readiness for Rare Neurological and Neuromuscular Diseases (U01)

A 22-Year Follow-up Study of Long-term Cardiac Outcome and Predictors of Survival in Friedreich Ataxia

FXN Promoter Silencing in the Humanized Mouse Model of Friedreich Ataxia

Delayed-onset Friedreich's ataxia revisited

Evidence for chromosome fragility at the frataxin locus in Friedreich ataxia

Friedreich ataxia in Norway - an epidemiological, molecular and clinical study

Investigation of mitochondrial DNA variations among Indian Friedreich's ataxia (FRDA) patients

Key Patent Granted For AAVLife’s Gene-Therapy Program to Treat Cardiomyopathy in Friedreich’s Ataxia

New funding opportunity announced by NINDS, NIH for neuroscience investigators. LOIs are due Sept 6, 2015 RFA-NS-16-001

Call for Abstracts due Friday, Aug. 7th, 2015

Mitigation of Myocardial Ischemia-Reperfusion Injury via HIF-1α-Frataxin Signalling

The Role of Frataxin in Doxorubicin Mediated Cardiac Hypertrophy

Regional Cerebral Disease Progression in Friedreich's Ataxia: A Longitudinal Diffusion Tensor Imaging Study

Gene Expression Profile in Peripheral Blood Cells of Friedreich Ataxia Patients

TORC1 Inhibition by Rapamycin Promotes Antioxidant Defences in a Drosophila Model of Friedreich's Ataxia

Ultra-structural hair alterations in Friedreich's ataxia: A scanning electron microscopic investigation

GAA triplet-repeats cause nucleosome depletion in human genome

Identification of telomere dysfunction in FA

Cardioprotective HIF-1α-frataxin signaling

New cellular model to follow FA development in time-resolved way

Novel aberrant genetic and epigenetic events in FA

Cardiomyopathy in FA: New biomarker for staging cardiac involvement

Turning E. coli into a Frataxin-Dependent Organism

Turning Yeast into a Frataxin-Independent Organism

Switching Frataxin Function by Single Amino Acid Substitution within [Fe-S] Cluster Assembly Scaffold

Sensorimotor skills in Mouse mutants frataxin-deficient in muscle

Iron-sulfur Assembly Complex Catalyzes [2Fe-2S] Clusters on ISCU2

Interaction of frataxin with IscU of Fe-S clusters

Mitoferrin modulates iron toxicity in a drosophila model of Friedreich´s ataxia

Diffusion tensor imaging and tract-based spatial statistics analysis in Friedreich's ataxia patients

Structural characterization of metal binding to a cold-adapted frataxin

Abnormal Cochlear Potentials in Friedreich's Ataxia Point to Disordered Synchrony of Auditory Nerve Fiber Activity

Selective observation of the disordered import signal of a globular protein by in-cell NMR: The example of frataxins

Sensorimotor skills in Fxn KO/Mck mutants deficient for frataxin in muscle

GSK Discovery Fast Track Challenge opening soon

Ferredoxin, in conjunction with NADPH and ferredoxin-NADP reductase, transfers electrons to the complex IscS/IscU to promote iron-sulfur cluster assembly

Milestones in Friedreich ataxia: more than a century and still learning

Very late-onset friedreich ataxia with laryngeal dystonia

International Congress on "Friedreich's Ataxia & DNA Structure in Health and Disease" to be held April 11-13, 2015 at the All India Institute of Medical Sciences, New Delhi, India

Frataxin inactivation leads to steroid deficiency in flies and human ovarian cells

Genome-Engineering Tools to Establish Accurate Reporter Cell Lines That Enable Identification of Therapeutic Strategies to Treat Friedreich's Ataxia

Mammalian frataxin directly enhances sulfur transfer of NFS1 persulfide to both ISCU and free thiols

"Both Sides of the Wheelchair": The Views of Inpiduals with, and Parents of Inpiduals with Friedreich Ataxia Regarding Pre-symptomatic Testing of Minors

Biological and clinical characteristics of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS) cohort: a cross-sectional analysis of baseline data

Friedreich's ataxia: the European consortium

Frataxin knockdown in human astrocytes triggers cell death and the release of factors that cause neuronal toxicity

Poor fluorodeoxyglucose uptake in myocardial viability study in nondiabetic Friedreich's ataxia patient

Monitoring Cardiac Function During Idebenone Therapy in Friedreich's Ataxia

p53 directly regulates the transcription of the human frataxin gene and its lack of regulation in tumor cells decreases the utilization of mitochondrial iron

Treatment for speech disorder in Friedreich ataxia and other hereditary ataxia syndromes

Cerebellar pathology in Friedreich's ataxia: Atrophied dentate nuclei with normal iron content

Open-label pilot study of interferon gamma-1b in Friedreich ataxia

An AAV9 coding for frataxin clearly improved the symptoms and prolonged the life of Friedreich ataxia mouse models

Probing the Kinetic Stabilities of Friedreich's Ataxia Clinical Variants Using a Solid Phase GroEL Chaperonin Capture Platform

Comparing speech characteristics in spinocerebellar ataxias type 3 and type 6 with Friedreich ataxia

Nasality in Friedreich ataxia

Overlapping Binding Sites of the Frataxin Homologue Assembly Factor

A longitudinal VBM study monitoring treatment with erythropoietin in patients with Friedreich ataxia

Iron-sulfur cluster biogenesis in mammalian cells: New insights into the molecular mechanisms of cluster delivery

Understanding the frustration arising from the competition between function, misfolding, and aggregation in a globular protein

Overlapping Binding Sites of the Frataxin Homologue Assembly Factor and the Heat Shock Protein 70 Transfer Factor on the Isu Iron-sulfur Cluster Scaffold Protein

Frataxin activates Fe-S cluster biosynthesis by facilitating sulfur transfer chemistry

A 7-year-old girl with hypertrophic cardiomyopathy and progressive scoliosis

Deferiprone in Friedreich ataxia: A 6-Month randomized controlled trial

Epigenetic promoter silencing in Friedreich ataxia is dependent on repeat length

Frataxin deficiency increases cyclooxygenase 2 and prostaglandins in cell and animal models of Friedreich's ataxia

Post-Doctoral Fellowship available for research on Friedreich's Ataxia

Human Frataxin Activates Fe-S Cluster Biosynthesis by Facilitating Sulfur Transfer Chemistry

Sensitivity of Spatiotemporal Gait Parameters in Measuring Disease Severity in Friedreich Ataxia

The role of frataxin in fission yeast iron metabolism: Implications for Friedreich's ataxia

Myelin paucity of the superior cerebellar peduncle in inpiduals with Friedreich ataxia: an MRI magnetization transfer imaging study

The Alteration of the C-terminal Region of Human Frataxin Distorts its Structural Dynamics and Function

Dysregulation of cellular iron metabolism in Friedreich ataxia: from primary iron-sulfur cluster deficit to mitochondrial iron accumulation

Low Bone Mineral Density in Friedreich Ataxia

Mitochondrial dysfunction induced by frataxin deficiency is associated with cellular senescence and abnormal calcium metabolism

Atypical Friedreich ataxia in patients with FXN p.R165P point mutation or comorbid hemochromatosis

Nicotinamide in Friedreich's ataxia: useful or not?

Epigenetic and neurological effects and safety of high-dose nicotinamide in patients with Friedreich's ataxia: an exploratory, open-label, dose-escalation study

Dentate nuclei T2 relaxometry is a reliable neuroimaging marker in Friedreich's ataxia

Pathophysiogical and therapeutic progress in Friedreich ataxia

Cell and gene therapy for Friedreich ataxia - progress to date

Saccade reprogramming in Friedreich ataxia reveals impairments in the cognitive control of saccadic eye movement

Frataxin deficiency in neonatal rat ventricular myocytes targets mitochondria and lipid metabolism

Altered Nucleosome Positioning at the Transcription Start Site and Deficient Transcriptional Initiation in Friedreich Ataxia

Frataxin Silencing Inactivates Mitochondrial Complex I in NSC34 Motoneuronal Cells and Alters Glutathione Homeostasis

Gene Expression Profiling of Mitochondrial Oxidative Phosphorylation (OXPHOS) Complex I in Friedreich Ataxia(FRDA) Patients

Base Excision Repair of Chemotherapeutically-Induced Alkylated DNA Damage Predominantly Causes Contractions of Expanded GAA Repeats Associated with Friedreich's Ataxia

Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich's ataxia

Rescue of the Friedreich Ataxia Knockout Mutation in Transgenic Mice Containing an FXN-EGFP Genomic Reporter

Mitochondrial iron-sulfur cluster dysfunction in neurodegenerative disease.

Generation and Characterisation of Friedreich Ataxia YG8R Mouse Fibroblast and Neural Stem Cell Models

Edison Shedding Light on Energy Production in Diseased Cells

Safety and tolerability of carbamylated erythropoietin in Friedreich's ataxia

Molecular and clinical investigation of Iranian patients with Friedreich ataxia

Kinetics of folding of frataxin

Foundation-industry relationships - a new business model joint-venture philanthropy in therapy development

Usefulness of frataxin immunoassays for the diagnosis of Friedreich ataxia

Beyond loss of frataxin: the complex molecular pathology of Friedreich ataxia

Frataxin-bypassing Isu1: characterization of the bypass activity in cells and mitochondria

The kinetics of folding of frataxin

HFE p.C282Y heterozygosity is associated with earlier disease onset in Friedreich ataxia

Dysphagia and swallowing-related quality of life in Friedreich ataxia

Modeling of Friedreich ataxia-related iron overloading cardiomyopathy using patient-specific-induced pluripotent stem cells

Apoptotic cell death and altered calcium homeostasis caused by frataxin depletion in dorsal root ganglia neurons can be prevented by BH4 domain of Bcl-xL protein

Frataxin directly stimulates mitochondrial cysteine desulfurase by exposing substrate-binding sites

Frataxin mRNA Isoforms in FRDA Patients and Normal Subjects: Effect of Tocotrienol Supplementation

Methylene blue rescues heart defects in a Drosophila model of Friedreich's ataxia

Metabolic remodeling in frataxin-deficient yeast is mediated by Cth2 and Adr1

Cognitive Deficits In Friedreich Ataxia Correlate with Micro-structural Changes in Dentatorubral Tract

EFACTS - European Friedreich's Ataxia Consortium for Translational Studies

FARA, Ataxia UK and GoFAR invite proposals for projects to develop biomarkers by non-invasive approaches

White Matter Changes in Patients with Friedreich Ataxia after Treatment with Erythropoietin

Friedreich Ataxia Patient Tissues Exhibit Increased 5-Hydroxymethylcytosine Modification and Decreased CTCF Binding at the FXN Locus

A Potential New Therapeutic Approach for Friedreich Ataxia: Induction of Frataxin Expression With TALE Proteins

Molecular and Functional Alterations in a Mouse Cardiac Model of Friedreich Ataxia

Quantitative profiling and identification of differentially expressed plasma proteins in friedreich's ataxia

Induced Pluripotent Stem Cells from Friedreich Ataxia Patients Fail to Upregulate Frataxin During In Vitro Differentiation to Peripheral Sensory Neurons

Ferredoxin Competes with Bacterial Frataxin in Binding to the Desulfurase IscS

His86 from the N-Terminus of Frataxin Coordinates Iron and Is Required for Fe-S Cluster Synthesis

Friedreich Ataxia: Executive Control Is Related to Disease Onset and GAA Repeat Length

Biochemistry of cardiomyopathy in the mitochondrial disease Friedreich's ataxia

The role of the N-terminal tail for the oligomerization, folding and stability of human frataxin

Neurodegeneration in Friedreich's Ataxia: From Defective Frataxin to Oxidative Stress

The Gait Variability Index: A new way to quantify fluctuation magnitude of spatiotemporal parameters during gait

Diagnosis and Genetic Counseling for Friedreich's Ataxia: A time for consideration of TP-PCR in an Indian Setup

Perceived Effectiveness and Barriers to Physical Therapy Services for Families and Children With Friedreich Ataxia

Stem Cells from Wildtype and Friedreich's Ataxia Mice Present Similar Neuroprotective Properties in Dorsal Root Ganglia Cells

Triple therapy with deferiprone, idebenone and riboflavin in Friedreich's ataxia - open-label trial

Fatigue is frequent and multifactorial in Friedreich's ataxia

Radial diffusivity in the cerebellar peduncles correlates with clinical severity in Friedreich ataxia

Triple Therapy with Darbepoetin Alfa, Idebenone, and Riboflavin in Friedreich's Ataxia: an Open-Label Trial

High-dose thiamine improves the symptoms of Friedreich's ataxia

Downstream MMR processing contributes to GAA·TTC repeat expansion

Cardiac remodeling of intercalated discs in Friedreich's ataxia

Therapeutic Strategies in Friedreich's ataxia

Frataxin Deficiency Leads to Reduced Expression and Impaired Translocation of NF-E2-Related Factor (Nrf2) in Cultured Motor Neurons

Cerebello-cerebral connectivity deficits in Friedreich ataxia

Mitochondrial Diseases of the Brain

Cis-silencing of PIP5K1B evidenced in Friedreich's ataxia patient cells results in cytoskeleton anomalies

Deferiprone and idebenone rescue frataxin depletion phenotypes in a Drosophila model of Friedreich's ataxia

Assessment of upper limb function in young Friedreich ataxia patients compared to control subjects using a new three-dimensional kinematic protocol

Epigenetics in Friedreich's Ataxia: Challenges and Opportunities for Therapy

The Molecular Basis of Iron-induced Oligomerization of Frataxin and the Role of the Ferroxidation Reaction in Oligomerization

Bilateral cochlear implantation in Friedreich's ataxia: A case study

A Dynamic Model of Proteins that Form the Initial Iron-Sulfur Cluster Biogenesis Machinery in Yeast Mitochondria

Friedreich's ataxia-associated GAA repeats induce replication-fork reversal and unusual molecular junctions

Impact of Friedreich's Ataxia on health-care resource utilization in the United Kingdom and Germany

Effects of alkyl side chain modification of coenzyme Q10 on mitochondrial respiratory chain function and cytoprotection

The Reciprocal Cerebellar Circuitry in Human Hereditary Ataxia

Complexes between two GAA Repeats within DNA introduced into Cos-1 cells

Heterochromatinization induced by GAA-repeat hyperexpansion in Friedreich's ataxia can be reduced upon HDAC inhibition by Vitamin B3

A Dynamic Model of the Proteins that Form the Initial Iron-Sulfur Cluster Biogenesis Machinery in Yeast Mitochondria

Excessive motor overflow reveals abnormal inter-hemispheric connectivity in Friedreich ataxia

Very late-onset Friedreich ataxia: later than life expectancy?

Cognition in Late-Onset Friedreich Ataxia

Pharmacological Screening Using an FXN-EGFP Cellular Genomic Reporter Assay for the Therapy of Friedreich Ataxia

Friedreich ataxia: neuropathology revised

Genetic Variations Creating MicroRNA Target Sites in the FXN 3′-UTR Affect Frataxin Expression in Friedreich Ataxia

Frataxin deficiency leads to defects in expression of antioxidants and Nrf2 expression in dorsal root ganglia of the Friedreich's ataxia YG8R mouse model

Friedreich Ataxia: Dysarthria Profile and Clinical Data

7th International Conference on Iron-Sulfur Cluster Biogenesis and Regulation May 20-24, 2013

Missense Mutations Linked to Friedreich Ataxia Have Different but Synergistic Effects on Mitochondrial Frataxin Isoforms

Mammalian frataxin controls sulfur production and iron entry during de novo Fe4S4 cluster assembly

Autophagy induction extends lifespan and reduces lipids content in response to frataxin silencing in C. elegans

Friedreich's ataxia with retained reflexes: a phenotype and genotype correlation

Substantia Nigra Echogenicity in Friedreich's Ataxia Patients

Clinical use of frataxin measurement in a patient with a novel deletion in the FXN gene

Synthesis and Biological Activities of N-(3-Carboxylpropyl)-5-amino-2-hydroxy-3-tridecyl-1,4-benzoquinone and Analogues

Genomic deletions and point mutations induced in Saccharomyces cerevisiae by the trinucleotide repeats (GAA*TTC) associated with Friedreich's ataxia

Identification of nonferritin mitochondrial iron deposits in a mouse model of Friedreich ataxia

Can telomere shortening in human peripheral blood leukocytes serve as a disease biomarker of Friedreich's Ataxia?

Induced pluripotent stem cell derived neurons and cardiomyocytes as a model for mitochondrial defects in Friedreich's ataxia

Role of DNA Polymerases in Repeat-Mediated Genome Instability

Cells Lacking Pfh1, a Fission Yeast Homolog of Mammalian Frataxin, Display Constitutive Activation of the Iron Starvation Response

Neuroanatomical Correlates of Depression in Friedreich's Ataxia: a Voxel-Based Morphometry Study

R- and S-Equol have equivalent cytoprotective effects in Friedreich's Ataxia

Letter by Peverill Regarding Article, "The Heart in Friedreich Ataxia: Definition of Cardiomyopathy, Disease Severity, and Correlation With Neurological Symptoms"

Pms2 Suppresses Large Expansions of the (GAA·TTC)(n) Sequence in Neuronal Tissues

Frataxin deficiency unveils cell-context dependent actions of insulin-like growth factor I on neurons

Auditory pathway changes mirror overall disease progress in individuals with Friedreich ataxia

Relation of Cytosolic Iron Excess to Cardiomyopathy of Friedreich's Ataxia

Binaural Speech Processing in Individuals with Auditory Neuropathy

Genome-wide Screen Identifies Pathways that Govern GAA/TTC Repeat Fragility and Expansions in Dividing and Nondividing Yeast Cells

EPI-743 Reverses the Progression of the Pediatric Mitochondrial Disease- Genetically Defined Leigh Syndrome

Heterotrifunctional chemical crosslinking mass spectrometry confirms physical interaction between human frataxin and ISU

Management and therapy for cardiomyopathy in Friedreich's ataxia

Analysis of the structural and mechanistic factors in antioxidants that preserve mitochondrial function and confer cytoprotection

Skeletal muscle involvement in friedreich ataxia and potential effects of recombinant human erythropoietin administration on muscle regeneration and neovascularization

Unanswered Questions in Friedreich Ataxia

Human Mesenchymal Stem Cells Increase Anti-oxidant Defences in Cells Derived from Patients with Friedreich's Ataxia

Role of mismatch repair enzymes in GAA-TTC triplet-repeat expansion in Friedreich's ataxia induced pluripotent stem cells (iPSCs)

Therapeutic Developments in Friedreich Ataxia

miR-886-3p Levels Are Elevated in Friedreich Ataxia

Rationale for the Development of 2-Aminobenzamide Histone Deacetylase Inhibitors as Therapeutics for Friedreich Ataxia

Cardiomyopathy in Friedreich Ataxia: Clinical Findings and Research

A functional MRI study of motor dysfunction in Friedreich's Ataxia

DNA triplex structures in neurodegenerative disorder, Friedreich's ataxia

DNA mismatch repair complex MutSbeta promotes GAA•TTC repeat expansion in human cells

Auditory Processing Deficits in Children With Friedreich Ataxia

Friedreich Ataxia Clinical Outcome Measures: Natural History Evaluation in 410 Participants

Clinical Monitoring in a Patient With Friedreich Ataxia and Osteogenic Sarcoma

Cross-Sectional Analysis of Electrocardiograms in a Large Heterogeneous Cohort of Friedreich Ataxia Subjects

Friedreich Ataxia: New Pathways

Cardiac Transplantation in Friedreich Ataxia

Clinical Features of Friedreich Ataxia

Novel Diagnostic Paradigms for Friedreich Ataxia

Clinical data and characterization of the liver conditional mouse model exclude neoplasia as a non-neurological manifestation associated with Friedreich's ataxia

Apn1 AP-endonuclease is essential for the repair of oxidatively damaged DNA bases in yeast frataxin-deficient cells

Complete FXN Deletion in a Patient with Friedreich's Ataxia

Longitudinal change in dysarthria associated with Friedreich ataxia: a potential clinical endpoint

Elevation of serum cardiac troponin I in a cross-sectional cohort of asymptomatic subjects with Friedreich ataxia

Ultrasound-guided nerve blocks in the Charcot-Marie-Tooth disease and Friedreich's ataxia

TALE proteins induced the expression of the frataxin gene

Friedreich's Ataxia Causes Redistribution of Iron, Copper, and Zinc in the Dentate Nucleus

Spinal Cord Atrophy Correlates with Disability in Friedreich's Ataxia

Human adipose stem cell-conditioned medium increases survival of Friedreich's ataxia cells submitted to oxidative stress

Decreased functional brain activation in Friedreich ataxia using the Simon effect task

Scoliosis in patients with Friedreich's ataxia

Estrogen Prevents Oxidative Damage to the Mitochondria in Friedreich's Ataxia Skin Fibroblasts

Abstracts to be presented at the American Academy of Neurology April 21-28

Retrospective study of the effects of inpatient rehabilitation on improving and maintaining functional independence in people with Friedreich ataxia

Interferon gamma upregulates frataxin and corrects the functional deficits in a Friedreich ataxia model

Cardiac Dysfunction Exacerbated by Endocrinopathies in Friedreich Ataxia: A Case Series

Is Friedreich ataxia an epigenetic disorder?

Epigenetics, epidemiology and mitochondrial DNA diseases

Erythropoietin in Friedreich ataxia: No effect on frataxin in a randomized controlled trial

Exonic Deletions of FXN and Early-Onset Friedreich Ataxia

Friedreich's Ataxia reveals a mechanism for coordinate regulation of oxidative metabolism via feedback inhibition of the SIRT3 deacetylase

Selecting and isolating colonies of human induced pluripotent stem cells reprogrammed from adult fibroblasts

Biogenesis of iron-sulfur clusters in mammalian cells: new insights and relevance to human disease

Understanding the genetic and molecular pathogenesis of Friedreich's ataxia through animal and cellular models

EDITORIAL: Cardiomyopathy in Friedreich's Ataxia: Exemplifying the Challenges Faced by the Cardiologists in the Management of Rare Diseases

Cognition in Friedreich Ataxia

The mismatch repair system protects against intergenerational GAA repeat instability in a Friedreich ataxia mouse model

Oxidative stress induces mitochondrial fragmentation in frataxin-deficient cells

Differentiating profiles of speech impairments in Friedreich's ataxia: a perceptual and instrumental approach

Effects of Friedreich's ataxia GAA repeats on DNA replication in mammalian cells

Chromatin changes in the development and pathology of the Fragile X-associated disorders and Friedreich ataxia

FXN methylation predicts expression and clinical outcome in friedreich ataxia (article)

Use of an Adaptive Study Design in Single Ascending-Dose Pharmacokinetics of A0001 (α-Tocopherylquinone) in Healthy Male Subjects

Substantia nigra hypoechogenicity in friedreich ataxia

The yeast metacaspase is implicated in oxidative stress response in frataxin-deficient cells

HSC20 interacts with Frataxin and is involved in iron-sulfur cluster biogenesis and iron homeostasis

A gene expression phenotype in lymphocytes from friedreich ataxia patients

A TAT-Frataxin fusion protein increases lifespan and cardiac function in a conditional Friedreich's Ataxia mouse model

Ophthalmic features of Friedreich ataxia

Exploring frataxin function

Hepatic mitochondrial dysfunction in Friedreich Ataxia

Primary and Secondary Drug Screening Assays for Friedreich Ataxia

Changes in mitochondrial glutathione levels and protein thiol oxidation in Δyfh1 yeast cells and the lymphoblasts of patients with Friedreich ataxia

MR spectroscopy and atrophy in Gluten, Friedreich's and SCA6 ataxias

Analysis of Echocardiograms in a Large Heterogeneous Cohort of Patients With Friedreich Ataxia

Annual change in Friedreich's ataxia evaluated by the scale for the assessment and rating of ataxia (SARA) is independent of disease severity

The effects of idebenone on mitochondrial bioenergetics

Specific Alterations of Carbohydrate Metabolism Are Associated With Hepatocarcinogenesis in Mitochondrially Impaired Mice

Oligomerization Propensity and Flexibility of Yeast Frataxin Studied by X-ray Crystallography and Small-Angle X-ray Scattering

Inactivation of mitochondrial aspartate aminotransferase contributes to the respiratory deficit of yeast frataxin-deficient cells

Friedreich's ataxia: the vicious circle hypothesis revisited

Initial Experience in the Treatment of Inherited Mitochondrial Disease with EPI-743

Mesenchymal stem cells restore frataxin expression and increase hydrogen peroxide scavenging enzymes in friedreich ataxia fibroblasts

Mutation in Fe-S scaffold Isu bypasses frataxin deletion

PEP-1-Frataxin Significantly Increases Cell Proliferation and Neuroblast Differentiation by Reducing Lipid Peroxidation in the Mouse Dentate Gyrus

The scale for the assessment and rating of ataxia correlates with dysarthria assessment in Friedreich's ataxia

The role of CyaY in iron sulfur cluster assembly on the E. coli IscU scaffold protein

A novel deletion-insertion mutation identified in exon 3 of FXN in two siblings with a severe Friedreich ataxia phenotype

Friedreich's Ataxia: a review from a cardiology perspective

The role of induced pluripotent stem cells in regenerative medicine: neurodegenerative diseases

The Fitts task reveals impairments in planning and online control of movement in Friedreich ataxia: reduced cerebellar-cortico connectivity?

Assessment of neurological efficacy of idebenone in pediatric patients with Friedreich's ataxia

Structure-function analysis of Friedreich's ataxia mutants reveals determinants for frataxin binding and activation of the Fe-S assembly complex

Rapamycin reduces oxidative stress in frataxin-deficient yeast cells

Articulatory Kinematics in the Dysarthria Associated with Friedreich's Ataxia

Evaluation of histone deacetylase inhibitors as therapeutics for neurodegenerative diseases

Overexpression of human and fly frataxins in Drosophila provokes deleterious effects at biochemical, physiological & developmental levels

Overexpression of human and fly frataxins in Drosophila provokes deleterious effects at biochemical, physiological and developmental levels

Long Range Regulation of Human FXN Gene Expression

Correlation of frataxin content in blood and skeletal muscle endorses frataxin as a biomarker in Friedreich ataxia

Neurodegeneration in friedreich's ataxia is associated with a mixed activation pattern of the brain. A fMRI study

Friedreich's ataxia variants I154F and W155R diminish frataxin-based activation of the iron-sulfur cluster assembly complex

Hyperexpansion of GAA repeats affects post-initiation steps of FXN transcription in Friedreich's ataxia

Gait Pattern in Inherited Cerebellar Ataxias

Differential Expression of PGC-1alpha and Metabolic Sensors Suggest Age-Dependent Induction of Mitochondrial Biogenesis in Friedreich Ataxia Fibroblasts

Mortality in Friedreich Ataxia

The cerebellar component of Friedreich's ataxia

Utilisation of Advance Motor Information is Impaired in Friedreich Ataxia

In children with Friedreich ataxia, muscle and ataxia parameters are associated

The Heart in Friedreich's Ataxia: Basic Findings and Clinical Implications

Effects of Erythropoietin on Frataxin Levels and Mitochondrial Function in Friedreich Ataxia - a Dose-Response Trial

Silencing of frataxin gene expression triggers a p53 dependent apoptosis in human neuron-like cells

Early Changes in Left Ventricular Long-Axis Function in Friedreich Ataxia: Relation with the FXN Gene Mutation and Cardiac Structural Change

Altered Zinc Transport Disrupts Mitochondrial Protien Processing/Import In Fragile X-Associated Tremor/Ataxia Syndrome

Idebenone Induces Apoptotic Cell Death in the Human Dopaminergic Neuroblastoma SHSY-5Y Cells

Silencing of frataxin gene expression triggers p53-dependent apoptosis in human neuron-like cells

Friedreich's ataxia: Past, present and future

Infectious delivery and long-term persistence of transgene expression in the brain by a 135-kb iBAC-FXN genomic DNA expression vector

NQO1-Dependent Redox Cycling of Idebenone: Effects on Cellular Redox Potential and Energy Levels

Detection of interruptions in the GAA trinucleotide repeat expansion in the FXN gene of Friedreich ataxia

A combined nucleic Acid and protein analysis in friedreich ataxia: implications for diagnosis, pathogenesis and clinical trial design

Brain diffusion-weighted imaging in Friedreich's ataxia

Cardiomyopathy of Friedreich's Ataxia: Use of Mouse Models to Understand Human Disease and Guide Therapeutic Development

Frataxin participates to the hypoxia-induced response in tumors

Quantification of Circulating Plasma DNA in Friedreich's Ataxia and Spinocerebellar Ataxia Types 2 and 12

Prolonged treatment with pimelic o-aminobenzamide HDAC inhibitors ameliorates the disease phenotype of a Friedreich ataxia mouse model

Impaired myocardial perfusion reserve & fibrosis in Friedreich ataxia: a mitochondrial cardiomyopathy with metabolic syndrome

Impaired inhibition of prepotent motor tendencies in Friedreich ataxia demonstrated by the Simon interference task

Mammalian Frataxin: An Essential Function for Cellular Viability through an Interaction with a Preformed ISCU/NFS1/ISD11 Iron-Sulfur Assembly Complex

The Mitochondrial Connection in Auditory Neuropathy

Divalent counterion-induced condensation of triple-strand DNA

Friedreich's Ataxia (GAA)(n)-(TTC)(n) Repeats Strongly Stimulate Mitotic Crossovers in Saccharomyces cerevisae

Iron-dependent functions of mitochondria-relation to neurodegeneration

Blood cells from Friedreich ataxia patients harbor frataxin deficiency without a loss of mitochondrial function

Friedreich's ataxia: Pathology, pathogenesis, and molecular genetics

Role of transcript & interplay between transcription and replication in triplet-repeat instability in mammalian cells

Preventing the ubiquitin/proteasome-dependent degradation of frataxin, the protein defective in Friedreich's Ataxia

2010 FARA Scientific News

Generation of Induced Pluripotent Stem Cell Lines from Friedreich Ataxia Patients

Impaired myocardial perfusion reserve and fibrosis in Friedreich ataxia: a mitochondrial cardiomyopathy with metabolic syndrome

Improved Histone Deacetylase Inhibitors as Therapeutics for the Neurodegenerative Disease Friedreich's Ataxia: A New Synthetic Route

The Neuropathology of Late-Onset Friedreich's Ataxia

Axial diffusivity is increased in the degenerating superior cerebellar peduncles of Friedreich's ataxia

Repeat expansion affects both transcription initiation and elongation in friedreich ataxia cells

Activation of mitochondrial energy metabolism protects against cardiac failure

Feasibility of Implantable Cardioverter Defibrillator Treatment in Five Patients With Familial Friedreich's Ataxia-A Case Series

Superior Cerebellar Peduncle Atrophy in Friedreich's Ataxia Correlates with Disease Symptoms

The Test of Everyday Attention Reveals Significant Sustained Volitional Attention and Working Memory Deficits in Friedreich Ataxia

Whole-body vibration alters blood flow velocity and neuromuscular activity in Friedreich's ataxia

Epoetin alfa increases frataxin production in Friedreich's ataxia without affecting hematocrit

Spectral measures of the effects of Friedreich's ataxia on speech

Friedreich's Ataxia Induced Pluripotent Stem Cells Model Intergenerational GAA·TTC Triplet Repeat Instability

Getting to the core of repeat expansions by cell reprogramming

Exploration of transitional life events in individuals with Friedreich ataxia: Implications for genetic counseling

Electrophysiology of Respiratory Chain Complexes and ADP/ATP Exchanger in Native Mitochondrial Membranes

Normal and Friedreich ataxia cells express different isoforms of frataxin with complementary roles in iron-sulfur cluster assembly

Structural bases for the interaction of frataxin with the central components of iron-sulphur cluster assembly

Human frataxin is an allosteric switch that activates the Fe-S cluster biosynthetic complex

Combined Therapy with Idebenone and Deferiprone in Patients with Friedreich's Ataxia

Restless Legs and Substantia Nigra Hypoechogenicity are Common Features in Friedreich's Ataxia

Successful treatment of auditory perceptual disorder in individuals with Friedreich ataxia

Role of transcript and interplay between transcription and replication in triplet-repeat instability in mammalian cells

Current and emerging treatment options in the management of Friedreich ataxia

The Friedreich's Ataxia protein frataxin modulates DNA base excision repair in prokaryotes and mammals

Molecular Details of the Yeast Frataxin-Isu1 Interaction during Mitochondrial Fe-S Cluster Assembly

Protective effects of transduced PEP-1-Frataxin protein on oxidative stress-induced neuronal cell death

Protocol proposal for Friedreich ataxia molecular diagnosis using fluorescent and triplet repeat primed polymerase chain reaction

1H MR Spectroscopy in Friedreich's Ataxia and Ataxia with Oculomotor Apraxia Type 2

Defects in Mitochondrial Axonal Transport and Membrane Potential without Increased Reactive Oxygen Species Production in a Drosophila Model of Friedreich Ataxia

Prospects for the use of artificial chromosomes and minichromosome-like episomes in gene therapy

Synthesis and characterization of mitoQ and idebenone analogues as mediators of oxygen consumption in mitochondria

Expression of Human Frataxin Is Regulated by Transcription Factors SRF and TFAP2

A Phase 3, Double-blind, Placebo-Controlled Trial of Idebenone in Friedreich Ataxia

Exercise Capacity and Idebenone Intervention in Children and Adolescents With Friedreich Ataxia

A rapid, noninvasive immunoassay for frataxin: Utility in assessment of Friedreich ataxia

Evaluation of neuropsychological functions in patients with Friedreich ataxia before and after cognitive therapy

Transposon Tn7 Preferentially Inserts into GAA-TTC Triplet Repeats under Conditions Conducive to Y-R-Y Triplex Formation

Frataxin & mitochondrial Fe-S cluster biogenesis

Frataxin and mitochondrial Fe-S cluster biogenesis

Variations of frataxin protein levels in normal individuals

Mitochondrial iron trafficking and the integration of iron metabolism between the mitochondrion and cytosol

Human iron-sulfur cluster assembly, cellular iron homeostasis and disease

Analysis of the factors influencing the cardiac phenotype in Friedreich's ataxia

Ocular Motor Fixation Deficits in Friedreich Ataxia

Altered lipid metabolism in a Drosophila model of Friedreich's ataxia

Carbamylated erythropoietin increases frataxin independent from the erythropoietin receptor

Nitric oxide & frataxin: two players contributing to maintain cellular iron homeostasis

Dysarthria in Friedreich's Ataxia: A Perceptual Analysis

Iron redistribution as a therapeutic strategy for treating diseases of localized iron accumulation

Understanding the molecular mechanisms of Friedreich Ataxia to develop therapeutic approaches

Influence of Genetic Factors on Left Ventricular Structure and Function in Friedreich's Ataxia

Therapeutic use of coenzyme Q(10) and coenzyme Q(10)-related compounds and formulations

Intermediate-dose idebenone and quality of life in Friedreich ataxia

Health-related quality of life in children with Friedreich ataxia

Long intronic GAA repeats causing Friedreich ataxia impede transcription elongation

North and South Indian Populations Share a Common Ancestral Origin of Friedreich's Ataxia but Vary in Age of GAA Repeat Expansion

PGC-1alpha Down-Regulation Affects the Antioxidant Response in Friedreich's Ataxia

Pathology and pathogenesis of sensory neuropathy in Friedreich's ataxia

Friedreich ataxia presenting as sudden cardiac death in childhood: Clinical, genetic and pathological correlation, with implications for genetic testing and counselling

Does oxidative stress contribute to the pathology of Friedreich's ataxia? A radical question

Evidence that yeast frataxin is not an iron storage protein In Vivo

Prevalence of interatrial block in patients with Friedreich's Ataxia

A role for p53 in mitochondrial stress response control of longevity in C. elegans

Automatic method of pause measurement for normal and dysarthric speech

Atypical, perhaps under-recognized? An unusual phenotype of Friedreich ataxia

FRIEDREICH'S ATAXIA: MOLECULAR MECHANISMS, REDOX CONSIDERATIONS AND THERAPEUTIC OPPORTUNITIES

Frataxin, a molecule of mystery: trading stability for function in its iron-binding site

Flavin Adenine Dinucleotide Rescues the Phenotype of Frataxin Deficiency

Two New Pimelic Diphenylamide HDAC Inhibitors Induce Sustained Frataxin Upregulation in Cells from Friedreich's Ataxia Patients and in a Mouse Model

A high throughput electrochemiluminescence assay for the quantification of frataxin protein levels

Altered Gene Expression and DNA Damage in Peripheral Blood Cells from Friedreich''s Ataxia Patients: Cellular Model of Pathology

Measuring the rate of progression in Friedreich ataxia: Implications for clinical trial design

Molecular control of the cytosolic aconitase/IRP1 switch by extramitochondrial frataxin

2009 FARA Scientific News

Iron binding activity in yeast frataxin entails a trade off with stability in the alpha1/beta1 acidic ridge region

Clinical measures of dysarthria in Friedreich Ataxia

The ins and outs of mitochondrial iron-loading: the metabolic defect in Friedreich's ataxia

Yeast frataxin mutants display decreased superoxide dismutase activities crucial to promote protein oxidative damage

Impairment in motor reprogramming in Friedreich ataxia reflecting possible cerebellar dysfunction

Epigenetic Silencing in Friedreich Ataxia Is Associated with Depletion of CTCF (CCCTC-Binding Factor) and Antisense Transcription

Progressive GAA·TTC Repeat Expansion in Human Cell Lines

Auditory Perception in Individuals with Friedreich's Ataxia

Frataxin interacts with Isu1 through a conserved tryptophan in its ß-sheet

Subclinical Myocardial Ischemia Without LV Hypertrophy in Friedreich's Ataxia

The N-terminus of mature human frataxin is intrinsically unfolded

A comparison of three measures of upper limb function in Friedreich ataxia

Characterizing gait, locomotor status, and disease severity in children and adolescents with Friedreich ataxia

Diverse effects in Friedreich's ataxia place PGC-1alpha center-stage

Chemical probes identify a role for histone deacetylase 3 in Friedreich's ataxia gene silencing

Friedreich's ataxia: Oxidative stress and cytoskeletal abnormalities

Histone Deacetylase Inhibitors and Neurodegenerative Disorders: Holding the Promise

The dorsal root ganglion in Friedreich's ataxia

Limitations in a frataxin knockdown cell model for Friedreich ataxia in a high-throughput drug screen.

Elucidation of the mechanism of mitochondrial iron loading in Friedreich's ataxia by analysis of a mouse mutant

The first cellular models based on frataxin missense mutations that reproduce spontaneously the defects associated with Friedreich ataxia

Frataxin deficiency induces schwann cell inflammation and death.

Comparison of three clinical rating scales in Friedreich ataxia (FRDA)

Nitric oxide and frataxin: two players contributing to maintain cellular iron homeostasis

Subjective Improvement in Proprioception in 2 Patients With Atypical Friedreich Ataxia Treated With Varenicline (Chantix)

Large-scale expansions of Friedreich's ataxia GAA repeats in yeast

Novel antibody-based strategies for the rapid diagnosis of mitochondrial disease and dysfunction.

Functional genomic analysis of frataxin deficiency reveals tissue-specific alterations and identifies the PPAR{gamma} pathway as a therapeutic target in Friedreich's ataxia.

Friedreich's ataxia impact scale: A new measure striving to provide the flexibility required by today's studies.

PNA binding and its effect on in vitro transcription in friedreich's ataxia triplet repeats.

Diagnosis and treatment of Friedreich ataxia: a European perspective.

Peptide nucleic acid (PNA) binding and its effect on in vitro transcription in friedreich's ataxia triplet repeats.

Bacterial frataxin CyaY is the gatekeeper of iron-sulfur cluster formation catalyzed by IscS.

A combined voxel-based morphometry and (1)H-MRS study in patients with Friedreich's ataxia.

Complex I and ATP content deficiency in lymphocytes from Friedreich's ataxia.

Pharmacotherapy for friedreich ataxia.

Integration of functional bacterial artificial chromosomes into human cord blood-derived multipotent stem cells.

Friedreich's Ataxia: From the (GAA)( n ) Repeat Mediated Silencing to New Promising Molecules for Therapy.

Overexpression of the yeast frataxin homolog (Yfh1): contrasting effects on iron-sulfur cluster assembly, heme synthesis and resistance to oxidative stress.

Impaired nuclear Nrf2 translocation undermines the oxidative stress response in friedreich ataxia.

Frataxin deficiency causes upregulation of mitochondrial Lon and ClpP proteases and severe loss of mitochondrial Fe-S proteins.

Idebenone in Friedreich's ataxia

In vivo imaging of pyrrole-imidazole polyamides with positron emission tomography

The in vivo mitochondrial two-step maturation of human frataxin

The longitudinal course of cardiomyopathy in Friedreich's ataxia during childhood

DNA methylation in intron 1 of the frataxin gene is related to GAA repeat length and age of onset in Friedreich’s ataxia patients

Urinary isoprostanes in Friedreich ataxia: lack of correlation with disease features

The MCK mouse heart model of Friedreich's ataxia

Drosophila Frataxin: An Iron Chaperone during Cellular Fe–S Cluster Bioassembly

Influence of Friedreich Ataxia GAA Noncoding Repeat Expansions on Pre-mRNA Processing

Recombinant human erythropoietin increases frataxin protein expression without increasing mRNA expression

Human Nbp35 is essential for both cytosolic iron-sulfur protein assembly and iron homeostasis

Health related quality of life measures in Friedreich Ataxia

Glutathione-dependent redox status of frataxin-deficient cells in a yeast model of Friedreich's ataxia

Deferiprone targets aconitase: implication for Friedreich's ataxia treatment

Dynamics, stability and iron-binding activity of frataxin clinical mutants

Speech perception ability in individuals with Friedreich ataxia

A cytosolic iron chaperone that delivers iron to ferritin

Lateral-flow immunoassay for the frataxin protein in Friedreich's ataxia patients and carriers

Novel, complex interruptions of the GAA repeat in small, expanded alleles of two affected siblings with late-onset Friedreich ataxia

Ancestral roles of eukaryotic frataxin

Protection against hydrogen peroxide-mediated cytotoxicity in Friedreich's ataxia fibroblasts using novel iron chelators of the 2-pyridylcarboxaldehyde isonicotinoyl hydrazone class

Iron-dependent regulation of frataxin expression: implications for treatment of Friedreich ataxia

Antioxidant use in Friedreich ataxia

HDAC Inhibitors Correct Frataxin Deficiency in a Friedreich Ataxia Mouse Model

Structural basis of the iron storage function of frataxin from single-particle reconstruction of the iron-loaded oligomer

Orthopedic shoes improve gait in Friedreich's ataxia: a clinical and quantified case study

Friedreich's ataxia and scoliosis: the experience at two institutions

Proteomic analysis of hearts from frataxin knockout mice

Binding of yeast frataxin to the scaffold for Fe-S cluster biogenesis, Isu

Brain white matter tracts degeneration in Friedreich ataxia

The Friedreich ataxia GAA repeat expansion mutation induces comparable epigenetic changes in human and transgenic mouse brain and heart tissues

The effects of frataxin silencing in HeLa cells are rescued by the expression of human mitochondrial ferritin

Repair of DNA double-strand breaks within the (GAA•TTC)n sequence results in frequent deletion of the triplet-repeat sequence

Phosphorus-31 two-dimensional chemical shift imaging in the myocardium of patients with late onset of Friedreich ataxia

Overexpression of frataxin in the mitochondria increases resistance to oxidative stress and extends lifespan in Drosophila

Drug Insight: antioxidant therapy in inherited ataxias

Redistribution of accumulated cell iron: a modality of chelation with therapeutic implications

Brain structural damage in Friedreich's ataxia

Vestibular, saccadic and fixation abnormalities in genetically confirmed Friedreich ataxia

Major targets of iron-induced protein oxidative damage in frataxin-deficient yeasts are magnesium-binding proteins

Idebenone treatment in paediatric and adult patients with Friedreich ataxia: long-term follow-up

DNA triplexes and Friedreich ataxia

Hydrogen peroxide scavenging rescues frataxin deficiency in a Drosophila model of Friedreich's ataxia

Call for Patients with Friedreich’s Ataxia

Targeting the gene in Friedreich Ataxia

Santhera Announces Start of US Phase III Clinical Trial

Mortality in Friedreich's Ataxia

Results Announced Phase I/II Study of Deferiprone

Moonlighting Enzyme Linked To Neurodegenerative Disease

Santhera Updates Development Strategy

NIH and Santhera Announce Positive Results of Study with SNT-MC17

Health: When Cells Stop Working

A Molecule That Binds GAA-TCC Triplet Repeat DNA Increases Frataxin Protein in Patient Cells

Edison Pharmaceuticals Announces FDA Grants EPI-A0001

Idebenone Phase II Trial Reaches Midpoint at National Institutes of Health

Austrian Lab Supported by FARA Publishes Exciting Findings

Santhera starts European Phase III Study with its Lead Compound SNT-MC17 (idebenone) in Friedreich’s Ataxia

Santhera and the NIH Collaborate to Evaluate SNT-MC17 in FA

NINDS Recruiting Patients for Phase 2 Idebenone Study

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