https://www.gosh.nhs.uk/our-research/our-research-infrastructure/nihr-great-ormond-street-hospital-brc/brc-news/?page=44

Biomedical Research Centre news

Accelerated approval granted for drug developed at GOSH

14 Dec 2016, 9:55 a.m.

The United States Food and Drug Administration (FDA) granted accelerated approval for a new medication to treat Duchenne muscular dystrophy (DMD). The drug, Eteplirsen, was developed by a UK consortium led by Novel Therapies Theme lead, Professor Francesc

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Investigation into the effectiveness of an extended gene panel for the diagnosis of complex neurometabolic phenotypes

14 Dec 2016, 9:45 a.m.

BRC-supported Dr Philippa Mills, Professor Peter Clayton and Professor Paul Gissen have led an investigation into the effectiveness of a gene panel, targeting 614 genes, in establishing a diagnosis for patients presenting with a wide array of neurometabol

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Genetic Code

North Thames recruitment to 100,000 genome project exceptionally strong for rare diseases

14 Dec 2016, 9:41 a.m.

The North Thames Genomic Medicine Centre’s (GMC) has recruited 5,200 genomes to the 100,000 Genomes project for rare diseases, making up around 28% of those recruited nationally.

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Impact of EU Referendum vote on gene and cell therapy in the UK

4 Oct 2016, 1:58 p.m.

In a new editorial published in Human Gene Therapy, leading researchers call for urgent action to ensure continued access to critical funding and ongoing collaborative opportunities with the broader scientific community in the European Union.

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Identification of biomarker which could aid risk stratification in Juvenile Dermatomyositis

16 Sep 2016, 11:12 a.m.

Research led by BRC-funded Professor Lucy Wedderburn has shown that muscle pathology and myositis-specific antibodies (MSA) predict the risk of remaining on treatment in Juvenile Dermatomyositis (JDM). These findings could aid risk stratification.

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