Archives

Today, Solid Biosciences released a letter to the Duchenne muscular dystrophy (DMD) community regarding the decision by the US Food and Drug Administration (FDA) to place the company’s IGNITE DMD…

#GivingTuesday, annually held immediately after the Black Friday and Cyber Monday holiday shopping days following Thanksgiving, falls late this year on Dec. 3. But here at MDA, we’re already planning…

Today, Roche Genentech released a letter to the Duchenne muscular dystrophy (DMD) community regarding its decision to discontinue its DMD clinical development program. The full letter follows. Letter to the…

At more than 150 of the leading healthcare institutions across the US, MDA Care Centers offer top clinical care to individuals living with muscular dystrophy, ALS, and other neuromuscular diseases.…

On Oct. 30, AveXis released a letter to the spinal muscular atrophy (SMA) community regarding a decision by the US Food and Drug Administration (FDA) to place a partial hold…

Several biotech and pharma companies presented interim results at the 24th Annual Congress of the World Muscle Society (WMS) held earlier this month in Copenhagen, Denmark. This roundup covers a…

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…

In July of this year, Capricor Therapeutics announced positive interim results from its phase 2 (HOPE-2) clinical trial evaluating CAP-1002, the company’s lead investigational therapy for Duchenne muscular dystrophy (DMD),…

Charles Thornton, MD, professor of Neurology at the University of Rochester, was awarded an MDA clinical research network grant (CRNG) totaling $1,118,673 over three years to continue to lead the…

Two years ago, I remember looking through the photos from the MDA Public Policy and Advocacy conference, excited to see so many in the neuromuscular disease community on Capitol Hill…