The prospect of widespread access to a life-changing drug for children with a rare muscular disorder is a step closer today after the United States Food and Drug Administration (FDA) granted accelerated approval for a new medication.
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).
The Somers CRF provides specialist day care accommodation for children and young people taking part in clinical research studies. This state of the art, purpose built facility is available to all GOSH/ICH staff undertaking clinical research, in particular early phase and experimental medicine trials.
Six-year-old Kamal is a patient who has taken part in a clinical trial at the Somers Clinical Research Facility (CRF), which provides specialist day care accommodation for children and young people taking part in clinical research study.
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.
Our long-term commitment to instigating, supporting and delivering cutting-edge research means there are a wide variety of research projects under way at Great Ormond Street Hospital (GOSH) at any given time.