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FARAFARA Cure FA

 

Scientific News

FARA funds research progress

In this section, you will find the most recent FA research publications, many of which are funded by FARA, as well as information on upcoming conferences and symposiums. You can search for articles by date using the archive box in the right hand column. To locate FARA Funded or Supported Research, click the hyperlink in the right hand column. You may also search for specific content using key words or phrases in the search button at the top right of your screen. Please be sure to visit other key research sections of our website for information on FARA’s Grant Program and the Treatment Pipeline.

 


 

Mitochondrial energy imbalance and lipid peroxidation cause cell death in Friedreich's ataxia

Friedreich's ataxia (FRDA) is an inherited neurodegenerative disease. The mutation consists of a GAA repeat expansion within the FXN gene, which downregulates frataxin, leading to abnormal mitochondrial iron accumulation, which may in turn cause changes in mitochondrial function. Although, many studies of FRDA patients and mouse models have been conducted in the past two decades, the role of frataxin in mitochondrial pathophysiology remains elusive. Are the mitochondrial abnormalities only a side effect of the increased accumulation of reactive iron, generating oxidative stress? Or does the progressive lack of iron-sulphur clusters (ISCs), induced by reduced frataxin, cause an inhibition of the electron transport chain complexes (CI, II and III) leading to reactive oxygen species escaping from oxidative phosphorylation reactions? To answer these crucial questions, we have characterised the mitochondrial pathophysiology of a group of disease-relevant and readily accessible neurons, cerebellar granule cells, from a validated FRDA mouse model.

Read the entire article HERE

Reata Pharmaceuticals, Inc. Announces Pricing of Initial Public Offering

IRVING, Texas, May 25, 2016 (GLOBE NEWSWIRE) -- Reata Pharmaceuticals, Inc. ("Reata"), a clinical-stage biopharmaceutical company, today announced the pricing of its initial public offering of 5,500,000 shares of its Class A common stock at $11.00 per share, before underwriting discounts and commissions. Reata has granted the underwriters a 30-day option to purchase up to 825,000 additional shares of its Class A common stock at the initial public offering price. The shares of Class A common stock are expected to begin trading on The NASDAQ Global Market on May 26, 2016, under the ticker symbol "RETA."

Read the entire Press Release HERE

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

Purkinje cell pathology is a common finding in a range of inherited and acquired cerebellar disorders, with the degree of Purkinje cell injury dependent on the underlying aetiology. Purkinje cells have an unparalleled resistance to insult and display unique regenerative capabilities within the central nervous system. Their response to cell injury is not typical of most neurons and likely represents both degenerative, compensatory and regenerative mechanisms. Here we present a pathological study showing novel and fundamental insights into Purkinje cell injury, remodelling and repair in Friedreich's ataxia; the most common inherited ataxia. Analysing post-mortem cerebellum tissue from patients who had Friedreich's ataxia, we provide evidence of significant injury to the Purkinje cell axonal compartment with relative preservation of both the perikaryon and its extensive dendritic arborisation.

Read the entire article HERE

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

Friedreich's ataxia (FRDA) is one of the most devastating childhood onset neurodegenerative disease affecting multiple organs in the course of progression. FRDA is associated with mitochondrial dysfunction due to deficit in a nuclear encoded mitochondrial protein, frataxin. Identification of disease-specific biomarker for monitoring the severity remains to be a challenging topic. This study was aimed to identify whether circulating cell-free nuclear DNA (nDNA) and mitochondrial DNA (mtDNA) in blood plasma can be a potential biomarker for FRDA. Clinical information was assessed using International Cooperative Ataxia Rating Scale and the disease was confirmed using Long-range PCR for GAA repeat expansion within the gene encoding frataxin. The frataxin expression was measured using Western blot. Plasma nDNA and mtDNA levels were quantified by Multiplex real-time PCR.

Read the entire article HERE

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

Also Licenses Rights to Patents and Pending Applications Covering Methods for Treating Friedreich's Ataxia With Interferon Gamma-1b; Company Currently Owns the Rights to Interferon Gamma-1b Under the Trade Name ACTIMMUNE(R) in the United States, Canada and Japan

DUBLIN, IRELAND -- (Marketwired) -- 05/19/16 -- Horizon Pharma plc (NASDAQ: HZNP) ("Horizon Pharma"), a biopharmaceutical company focused on improving patients' lives by identifying, developing, acquiring and commercializing differentiated and accessible medicines that address unmet medical needs, today announced that its affiliate has entered into a definitive agreement with Boehringer Ingelheim International GmbH ("Boehringer Ingelheim") to acquire the rights to interferon gamma-1b, which Boehringer Ingelheim commercializes under the trade names IMUKIN®, IMUKINE®, IMMUKIN® and IMMUKINE® in an estimated 30 countries primarily in Europe and the Middle East.

Read the entire Press Release HERE

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