https://www.gosh.nhs.uk/our-research/our-research-infrastructure/nihr-great-ormond-street-hospital-brc/brc-news/?page=47

Biomedical Research Centre news

Positive interim analysis of Phase 3 studies in spinal muscular atrophy

14 Dec 2016, 10:02 a.m.

Biogen and Ionis Pharmaceuticals announce positive results at interim analysis of two Phase 3 studies both trialling the drug nusinersen in patients with spinal muscular atrophy (SMA).

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

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

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.

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.