AavantiBio, a biotechnology company developing a gene-targeting therapy for Friedreich’s ataxia (FA), was awarded MDA Venture Philanthropy (MVP) funding totaling $1,076,232 to advance AavantiBio’s phase 2 clinical trial of a gene-replacement therapy for the disease. MVP is MDA’s drug development program, which is exclusively focused on funding the discovery and clinical application of treatments and . . .
Texas-based Reata Pharmaceuticals announced positive results of its pivotal phase 2 clinical trial (MOXIe) of omaveloxolone in patients with Friedreich’s ataxia (FA). Patients treated with omaveloxolone showed a statistically significant improvement in the modified Friedreich’s Ataxia Rating Scale mFARS (a scale used to measure neurological function) after 48 weeks of treatment compared to placebo, and . . .
Sanjay Bidichandani, MBBS, PhD, professor of Pediatrics at the University of Oklahoma Health Sciences Center, has an ongoing MDA research grant (totaling $300,000 over three years) to study the role of epigenetic silencing of the frataxin gene (FXN) in Friedreich’s ataxia (FA). (Epigenetic changes to the genome are factors that influence the activity of genes . . .
Spending time in our nation’s capital with dozens of other MDA families gave me a new sense of empowerment and taught me to apply a skill I’ve already honed in my personal life to the public policy arena. It was inspiring and exciting to see so many people come together in one place to do our part in making this country a better place for people who live with muscle-debilitating diseases.
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 . . .