Muscular Dystrophy News

MDA Awards $7M in Grants for 29 Disease-Targeting Research Projects

The Muscular Dystrophy Association (MDA) has awarded $7 million in grants to 29 top researchers for projects it says will potentially make a big impact on muscular dystrophy and other life-threatening diseases. The investment reflects the nonprofit organization’s plan to double spending on drug development and clinical trials by 2020. Each year,…

BioMarin Withdraws MAA for Kyndrisa (Drisapersen) in Europe

BioMarin Pharmaceutical Inc. has announced the withdrawal of its Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) for Kyndrisa (drisapersen), a drug developed for Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The decision follows deliberations at the May 2016 Committee for Medicinal Products for…

Duchenne MD Patients May Receive Investigational Drug at No Cost

Marathon Pharmaceuticals has announced the launch of ACCESS DMD, its expanded access program (EAP) to provide deflazacort to qualified patients with Duchenne muscular dystrophy (DMD) under the authorization of the U.S. Food and Drug Administration (FDA). Marathon Pharmaceuticals, LLC, a biopharmaceutical company that develops treatments for rare neurological diseases,…