https://www.gosh.nhs.uk/our-research/our-research-infrastructure/nihr-great-ormond-street-hospital-brc/brc-news/?page=53

Biomedical Research Centre news

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

9 Sep 2015, 4:41 p.m.

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)

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Mitochondria

Protein responsible for rare childhood disorder gives clues to common neurodegenerative conditions

9 Sep 2015, 4:10 p.m.

A new mutation in the protein STAT2 has been identified in patients with mitochondrial disease. These findings could also be beneficial for more common neurodegenerative diseases, including Alzheimer’s, Huntington’s and Parkinson’s diseases.

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