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Accelerated approval request for a new drug for a rare childhood muscle disorder

A drug for Duchenne Muscular Dystrophy (DMD), originally developed by BRC Theme Lead Professor Francesco Muntoni’s Consortium in the UK, has been filed by Sarepta Therapeutics for accelerated approval by the United States Food and Drug Adminstration (FDA).

Research and publications

The cleft team has a long history of clinical research and audit activity.  It is a multi-disciplinary service and the contributions have been from speech and language therapists, psychologists, audiological physicians, orthodontists, paediatricians, as well as surgeons.