ACTIMMUNE Phase 3 Trial in Friedreich’s Ataxia Disappoints

Results from the STEADFAST phase 3 clinical trial in Friedreich’s ataxia (FA) failed to show that the drug ACTIMMUNE® (interferon gamma-1b) was effective against any of the study’s disease measurements, according to Horizon Pharma. The study hoped to slow disease progression as measured by a functional rating scale assessing capacities such as speech, ability to . . .

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Five Questions with Friedreich’s Ataxia Researcher Michael Huang

Michael Huang, the NHMRC Peter Doherty Postdoctoral Fellow in the department of pathology, Bosch Institute, at the University of Sydney, Australia, was awarded an MDA development grant totaling $177,100 over a period of three years to explore how deficiency of the frataxin protein in Friedreich’s ataxia (FA) may alter the function of cellular power supplies . . .

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MDA Grants Work to Find Breakthroughs Across Diseases

Since its inception, MDA has invested more than $1 billion in neuromuscular disease research to uncover new treatments and cures. In 2016 alone, MDA awarded 66 new research, development and research infrastructure grants with a total funding commitment of more than $17 million. These and other MDA grants fund research projects designed to uncover the . . .

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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 . . .

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Michigan State Goodwill Ambassador

Matthew Bishop, Age 12 New Lothrop, MI Home and Family Matt lives with his parents, Colleen and Thomas, and his younger sister, Emma. The family also has a yellow lab named Molly and a cat named Sylvie. Diagnosis When Matt was 7, he was diagnosed with Friedreich’s Ataxia by the MDA Clinic at Children’s Hospital . . .

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South Dakota State Goodwill Ambassador

Tysen Cook, Age 16 Watertown, SD Home and Family Tysen is the son of Nicole Cook-Rieffenberger and Justin Ward. Tysen lives with his mother, step-father-to-be, Brad, and 3 brothers, Braxtyn, Camdyn and Cruz. Diagnosis Tysen was diagnosed with Friedreich’s Ataxia (FA). FA’s major neurological symptoms include muscle weakness and ataxia, a loss of balance and . . .

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

Alliance is part of an MDA initiative to establish new collaborative partnerships CHICAGO, September 2, 2015 – The Muscular Dystrophy Association (MDA) and the Friedreich’s Ataxia Research Alliance (FARA) today announced a partnership that will pave the way for advancements in Friedreich’s Ataxia (FA) research, therapeutic development and clinical care, thus deepening understanding of the . . .

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