The FA CollaMap

The FA CollaMap is an intuitive, up-to-date, collaborative map of all we know about Friedreich’s ataxia. It contains compressively sourced, neutrally described knowledge about FA, spanning basic, translational, and clinical science. This knowledge is being enriched by presenting gaps in our understanding of the disease and open questions (and ideally suggestions on how to tackle them), debates on controversial topics, ongoing research around the world, and information on current and past clinical trials.

The FA CollaMap is a living thing and can be edited by all users. After account registration and approval, anyone can contribute with knowledge and to the discussion.

Preclinical Research Tools & Resources

PRECLINICAL RESEARCH RESOURCE

Cell Line & Tissue Repositories

FARA has helped establish repositories to provide investigators access to a variety of primary cells and cell lines, biofluids, and tissues from FA patients.

PRECLINICAL RESEARCH RESOURCE

Mouse Models

FA mouse models with various degrees of frataxin protein reduction and phenotype severity have been developed.

PRECLINICAL RESEARCH RESOURCE

Expression Array and other -omic Data

Several transcriptomic, proteomic, and metabolomic studies using patient cells, cell lines, and animal models have been published.

PRECLINICAL RESEARCH RESOURCE

Assays for Measuring Frataxin Protein

Sensitive, accurate, and quantitative measurement of frataxin is an important biomarker in drug development and several assays have been developed for different contexts of use, both in vitro and in vivo.

PRECLINICAL RESEARCH RESOURCE

Assays to Measure Downstream Effects of Frataxin Deficiency

Consequences of reduced FXN expression and function include the activity of various Iron-Sulfur Cluster containing proteins, assays for metabolic changes, oxidative damage, and mitochondrial function deficit.

Clinical Research Tools & Resources

CLINICAL RESEARCH RESOURCE

Biomarkers and Clinical Assessments

Provided here is a detailed listing of biomarkers and clinical outcome assessments critical for the development of therapeutics for FA.

CLINICAL RESEARCH RESOURCE

Time to Diagnosis of FA

A meta-analysis of three data sets evaluating time to diagnosis of FA.

CLINICAL RESEARCH RESOURCE

Common Data Elements

The Friedreich's Ataxia (FA) Common Data Elements (CDEs) were developed by an NIH/NINDS working group of experts to standardize the collection, analysis, and sharing of data across various clinical studies focused on FA.

CLINICAL RESEARCH RESOURCE

FA Clinical Datasets

Through a collaborative partnership with the Rare Diseases Cures Accelerator Data and Analytic Platform (RDCA-DAP) of the Critical Path Institute (C-Path), FARA has curated and standardized multiple FA clinical trial and natural history data sets into CDISC format and made this data publicly available to qualified researchers. Our goal is to catalyze the sharing of data and knowledge to advance drug development for FA.

Clinical Data Collection Worksheets & Assessments

CLINICAL RESEARCH RESOURCE

FARS/mFARS Administration Instructions

This instruction document details how to administer Friedreich’s Ataxia Rating Scale (FARS), which is a neurological rating scale developed to measure neurological function in FA. The modified FARS (mFARS) has better psychometric properties and shortens the bulbar subscale.

CLINICAL RESEARCH RESOURCE

mFARS Info Sheet

This one-page info sheet provides a summary-level overview of the modified Friedreich’s Ataxia Rating Scale (mFARS).

CLINICAL RESEARCH RESOURCE

mFARS Data Collection Worksheet (Scoring with Instructions)

This document can be used to collect the data relevant to scoring for the modified Friedreich’s Ataxia Rating Scale (mFARS) and includes instructions.

CLINICAL RESEARCH RESOURCE

mFARS Data Collection Worksheet (Scoring Only)

This document can be used to collect the data relevant to scoring for the modified Friedreich’s Ataxia Rating Scale (mFARS).

CLINICAL RESEARCH RESOURCE

Functional Disability Staging (FDS) Data Collection Worksheet

This document can be used to collect Functional Disability Staging (FDS) score. This FDS scale is used to indicate the impact of FA on function.

CLINICAL RESEARCH RESOURCE

FA-ADL (English) Data Collection Worksheet

This document can be used to collect the FA Activities of Daily Living (ADL). The FA ADL is a short assessment of functional abilities in patients with FA.

CLINICAL RESEARCH RESOURCE

SARA Data Collection Worksheet

This document can be used to collect Scale for the Assessment and Rating of Ataxia (SARA). Endorsed by the European Reference Network for Rare Neurological Diseases (ERN-RND).

CLINICAL RESEARCH RESOURCE

SARA Training Tool – Ataxia Global Initiative (AGI)

Scale for the Assessment and Rating of Ataxia (SARA) Training Tool.