Charles Moore,  —

Sarepta Therapeutics of Cambridge, Massachusetts, reports it has been notified by the Food and Drug Administration (FDA) that the agency was unable to complete its review of Sarepta’s New Drug Application (NDA) for the Duchenne muscular dystrophy (DMD) drug candidate eteplirsen by the Prescription Drug User Fee Act (PDUFA)…

An expert panel convened by the U.S. Food and Drug Administration (FDA) recently voted against granting accelerated approval to the novel Duchenne muscular dystrophy (DMD) drug candidate, eteplirsen. The vote came after a review of clinical trial data and 11 hours of hearing testimony that focused on the drug’s efficacy,…

UPDATE: Since this story was filed, Reuters news service is reporting that even more quickly than we had anticipated was likely, the expert panel convened by the FDA this week to review clinical trial data pertaining to Sarepta’s Duchenne Muscular Dystrophy (DMD) drug candidate Eteplirsen has denied…

Ten-year-old Gabe Griffin of Alabama visited Washington, D.C., with his parents last month hoping to encourage Congress to pass a federal bill that could help accelerate the discovery of an effective treatment or cure for the rare and terminal form of muscular dystrophy he’s afflicted with. Duchenne…

Charles Thornton, M.D., the Saunders Family Distinguished Professor in Neuromuscular Research at the University of Rochester Medical Center’s (URMC) Center for Neural Development & Disease in Rochester, New York, has received a Javits Neuroscience Investigator Award from the National Institutes of Health’s National Institute of Neurological Disorders and…

Tens of thousands of businesses across the U.S. are participating in this year’s 34th annual Muscular Dystrophy Association (MDA) Shamrocks campaign to raise funds for research and support programs to help people with muscular dystrophy, amyotrophic lateral sclerosis (ALS), and other diseases that severely weaken muscle…

Capricor Therapeutics, Inc., a clinical-stage biotechnology company, has treated the first Duchenne muscular dystrophy patient with related cardiomyopathy with its drug candidate CAP-1002, and is continuing to enroll people in its HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) Phase I/II clinical trial (ClinicalTrials.gov Identifier: NCT02485938). CAP-1002 is a proprietary allogeneic,…

PTC Therapeutics, Inc. announced Wednesday that it has received a Refuse to File letter from the U.S. Food and Drug Administration (FDA) with regard to its New Drug Application (NDA) for ataluren (trade name, Translarna), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy…

One hundred and nine members of the U.S. House of Representatives signed a letter to the U.S. Food and Drug Administration (FDA) calling for the agency to immediately increase efforts to mobilize all available tools, resources, and authorities to advance new and more effective treatments for Duchenne muscular dystrophy. The bipartisan appeal…

In recognition that people with muscular dystrophy are living longer than even recently thought possible, the Muscular Dystrophy Association (MDA) has named a young man as its latest National Goodwill Ambassador — marking the first time in the program’s 64-year history that a child hasn’t been selected for that role.