The U.S. Food and Drug Administration (FDA) has accepted Marathon Pharmaceuticals’ New Drug Applications for deflazacort for the treatment of Duchenne muscular dystrophy (DMD) and granted Priority Review. Deflazacort, a glucocorticoid, works as an anti-inflammatory and immunosuppressant. In the United States, the drug is considered an investigational therapy, as it has not been approved by . . .
Researchers are looking for boys and men, ages 5 to 18 years, to participate in a natural history study that is designed to assess the potential of imaging techniques to monitor disease progression and serve as an outcome measure for clinical trials in Duchenne muscular dystrophy (DMD). Both healthy volunteers and individuals with DMD are . . .
In a joint statement today, Biogen and Ionis Pharmaceuticals announced encouraging interim results from the late-stage ENDEAR clinical trial to test the experimental drug nusinersen in infants with type 1 spinal muscular atrophy (SMA). Based on the results, investigators have stopped the trial to allow all participants to transition into an open-label trial called SHINE, . . .
MDA Amplify is a new mobile app that revolutionizes the way you — an MDA supporter, partner or family — can share MDA news on your own social media channels. To become an MDA Amplifier, download the MDA Amplify app from iTunes or Google Play store (it’s free!), connect your personal social media accounts, and . . .
At MDA, we are beginning 2016 with families at the heart of our mission – literally. The heart in the middle of our refreshed logo symbolizes the kids and adults living with muscular dystrophy, ALS and related life-threatening diseases we fight for every day. We also begin the year with new offerings and enhancements to . . .