Friedreich’s Ataxia News and Research

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Exercise has profound protective effect in mouse models of Friedreich's ataxia

A top exercise researcher at the University of Virginia School of Medicine is urging clinical trials of exercise in patients with Friedreich's ataxia after finding that physical activity has a "profound" protective effect in mouse models of the debilitating genetic disease.

16 Jun 2020

New molecular mechanism can reverse genetic defect responsible for Friedreich's ataxia

Scientists at Tufts University have identified a molecular mechanism that could reverse the genetic defect responsible for Friedreich's ataxia, a neurodegenerative disease that leaves its victims with difficulty walking, a loss of sensation in the arms and legs and impaired speech caused by degeneration of nerve tissue in the spinal cord.

6 Jan 2020

C-Path and FARA announce launch of Friedreich's Ataxia Integrated Clinical Database

Critical Path Institute's Data Collaboration Center and the Friedreich's Ataxia Research Alliance today announced the launch of the Friedreich's Ataxia Integrated Clinical Database.

28 Feb 2019

UCLA researchers reveal reversibility of Friedreich's ataxia in mouse models

In children and adults with Friedreich's ataxia, an inherited disease that causes damage to the nervous system, a loss of coordination typically progresses to muscle weakness and can involve vision, diabetes, and other problems over the course of several years.

20 Dec 2017

Early intervention may be crucial to successful treatment of Friedreich's ataxia

New research published today in Disease Models & Mechanisms indicates that early intervention should be a key target in the development of new therapeutics for Friedreich's ataxia, as current treatments may be administered too late to target the disease effectively.

8 Nov 2017

Synthetic RNA and DNA could reverse protein deficiency that causes Friedreich's ataxia

Researchers at UT Southwestern Medical Center have identified synthetic RNA and DNA that reverses the protein deficiency causing Friedreich's ataxia, a neurological disease for which there is currently no cure.

17 Feb 2016

Riluzole ‘a candidate treatment’ for cerebellar ataxia

Riluzole may be an effective treatment for cerebellar ataxia, say researchers who call for further studies to confirm their findings.

9 Sep 2015

USF, FARA to jointly host scientific symposium on Friedreich's ataxia

The University of South Florida will again bring together leading researchers and patients searching for a treatment for Friedreich's ataxia and related disorders at the seventh annual scientific symposium "Understanding Energy for A Cure." The symposium will be held 5 to 8:30 p.m., Thursday, Sept. 17, at the USF Marshall Student Center Ballroom, USF Cedar Circle, Tampa, FL 33620.

2 Sep 2015

Longtime allies team up to create Penn Medicine/CHOP Friedreich's Ataxia Center of Excellence

Three longtime allies have joined forces to create the new Penn Medicine/CHOP Friedreich's Ataxia Center of Excellence.

7 Mar 2014

Edison's vatiquinone receives Orphan Status for treatment of Friedreich's ataxia

Edison Pharmaceuticals today announced that the US Food and Drug Administration has granted Orphan Status to vatiquinone for the treatment of Friedreich's ataxia.

4 Feb 2014

FARA funds gene therapy-based research project to tackle Friedreich's ataxia

The gene therapy-based research project to tackle Friedreich's ataxia launched in November in labs at the Institute for Research in Biomedicine, in Barcelona, and the "Centro de Biolog-a Molecular Severo Ochoa", in Madrid, has received 100,000 US$ per year for two years from the Friedreich's Ataxia Research Alliance.

29 Jan 2014

Scientists join patients to address Friedreich's Ataxia

The Spanish Federation of Ataxia (FEDAES)—in representation of the GENEFA Platform for a Friedreich's Ataxia cure—, the Babel Family association for biomedical research into Friedreich's Ataxia, the "Centro de Biología Molecular Severo Ochoa" (CMBSO), and the Institute for Research in Biomedicine (IRB Barcelona) have signed an agreement through which these patients' associations will fund, by means of donations, a 3-year research project addressing Friedreich's Ataxia.

16 Nov 2013

Friedreich's Ataxia costs between £11,000 and £19,000 per person per year

What is the cost of rare diseases such as Friedreich's Ataxia? By analyzing direct and indirect costs of care research in BioMed Central's open access journal Orphanet Journal of Rare Diseases calculated that conservatively this rare disease costs between £11,000 and £19,000 per person per year.

28 Feb 2013

Edison Pharmaceuticals to commence EPI-743 phase 2B Friedreich's ataxia study

Edison Pharmaceuticals today announced the initiation of a phase 2B study entitled, "Safety and Efficacy Study of EPI-743 on Visual Function in Patients with Friedreich's Ataxia."

7 Jan 2013

Study discovers pathways that govern instability of GAA/TTC repeats

A study of more than 6,000 genes in a common species of yeast has identified the pathways that govern the instability of GAA/TTC repeats. In humans, the expansions of these repeats is known to inactivate a gene - FXN - which leads to Friedreich's ataxia, a neurodegenerative disease that is currently incurable. In yeast, long repeats also destabilize the genome, manifested by the breakage of chromosomes.

11 Sep 2012

Friedreich Ataxia Research Alliance, USF ARC to host ‘Understanding a Cure’ symposium

Scientists, clinicians and patients will gather at the University of South Florida's Center for Advanced Medical Learning and Simulation (CAMLS) in downtown Tampa Sept. 6 to share research insights and energize the search for a cure for Friedreich's ataxia and related disorders.

28 Aug 2012

FARA announces 2011 Ataxian Athlete Initiative grant recipients

The Friedreich's Ataxia Research Alliance (FARA), in partnership with The FA Project, Catrike, Anna Maria Oyster Bar, and The Melting Pot are pleased to announce Bill Ramsey, Tom Trovinger, Mary Vida, and Patrick Cogan as the 2011 Ataxian Athlete Initiative (AAI) grant recipients.

10 Oct 2011

ViroPharma licenses worldwide rights from Intellect Neurosciences to OX1 for treatment of FA

ViroPharma Incorporated today announced the license of worldwide rights from Intellect Neurosciences, Inc. to its clinical stage drug candidate, OX1, being developed for the treatment of Friedreich's Ataxia (FA), a rare, hereditary, progressive neurodegenerative disease.

1 Oct 2011

MDA announces $13.7M in grants to 40 new muscular dystrophy research initiatives

The Muscular Dystrophy Association today announced funding, totaling $13.7 million, for 40 new research initiatives targeting nearly two dozen progressive neuromuscular diseases.

24 Aug 2011

Repligen first quarter total revenue increases 9% to $7,654,000

Repligen Corporation today reported results for the first quarter of fiscal year 2012, ended June 30, 2011.

27 Jul 2011