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£75,000 boost for Duchenne Muscular Dystrophy gene therapy research

GOSH Biomedical Research Centre researcher Dr John Counsell has been awarded up to £75,000 of funding from the UCL Therapeutic Acceleration Support (TAS) Fund for a study exploring the use of novel vectors in gene therapy for Duchenne Muscular Dystrophy (DMD).

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).

Cannabidiol trial shows reduction in seizures for children with Dravet syndrome

A trial conducted in Europe and the USA has shown that cannabidiol – a drug derived from cannabis but with the psycho-active elements removed – reduces seizures in children with a form of drug resistant epilepsy, known as Dravet syndrome.

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