Friedreich’s Ataxia (FA)

MDA and FARA Partner to Advance Treatments and Care for Friedreich’s Ataxia

FA - Sanjay Bidichandani, M.B.B.S., Ph.D.

CMRI Claire Gordon Duncan Chair in Genetics and Professor of Pediatrics Sanjay Bidichandani, at University of Oklahoma Health Sciences Center in Oklahoma City, was awarded an MDA research grant totaling $300,000 over three years to address clinically and scientifically important questions regarding the use of existing and novel HDAC inhibitors to increase levels of the frataxin protein in Friedreich’s ataxia (FA).

New Forms of Genetic Testing Improve Diagnosis, Raise Questions

“Knowing, if not all, is almost all,” said Matthew Harms, a neurologist and neurophysiologist from Washington University in St. Louis, in his presentation on genetic testing for neuromuscular disorders at the 2014 MDA Clinical Conference, held in Chicago March 16-19.

FA: BioMarin To Develop Former Repligen Drugs

FA Drug RG2833 Moves from Repligen to BioMarin

Friedreich's Ataxia Registry Open for Enrollment

The Coordination of Rare Diseases at Sanford (CoRDS) national rare disease registry is now hosting an ataxia patient registry for people with Friedreich's ataxia (FA) or other disorders classified as ataxias (conditions that cause problems with balance or coordination).

Study Suggests Sleep-Disordered Breathing Common in FA

Results from a study conducted in Australia show that a nighttime respiratory problem called obstructive sleep apnea occurs more frequently in people with Friedreich's ataxia (FA) than in the general population.

FA: RG2833 Is Well-Tolerated and Increases Frataxin Gene Activity

Interim results from a phase 1 clinical trial of RG2833 in people with Friedreich’s ataxia (FA) show that the experimental drug is well-tolerated, and that it appears to increase the activity of the gene for the frataxin protein.

‘Focused, Intense’ MDA Conference Advances Neuromuscular Disease Research

Turning neuromuscular disease research into treatments as quickly and effectively as possible was the overarching theme of dozens of formal presentations, nearly 200 scientific posters, and countless informal conversations at the MDA Scientific Conference, April 21-24.

A palpable sense of excitement pervaded the sold-out event thanks to the unprecedented number of experimental treatments in clinical trials for neuromuscular diseases, and the unique opportunity the conference provided for information-sharing and collaboration among scientific professionals from many disciplines.

MDA Scientific Conference To Emphasize Therapy Development

The Muscular Dystrophy Association’s annual conference being held in Washington, D.C., on April 21-24, 2013, is centered on the theme Therapy Development for Neuromuscular Diseases: Translating Hope into Promise.

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