https://www.gosh.nhs.uk/our-research/our-research-infrastructure/nihr-great-ormond-street-hospital-brc/brc-news/?page=62

Biomedical Research Centre news

Clinical trial for a new stem-cell based therapy for a rare skin condition proves promising

9 Sep 2015, 4:52 p.m.

Promising findings from a trial for a new stem-cell based therapy for a rare skin condition have been published in the Journal of Investigative Dermatology. The study, which involved intravenous injections of stem-cells, has led to an improvement in the q

Funding provided to develop new therapy for acute liver failure

9 Sep 2015, 4:50 p.m.

BRC-supported researcher Dr Kevin Mills has been awarded funding from the NIHR to investigate the regenerative ability of the liver following acute liver failure (ALF). If successful, the team hope that their research will lead to a novel therapy that cou

Novel mutation in LHX4 leads to a severe form of hypopituitarism

9 Sep 2015, 4:47 p.m.

Researchers at GOSH and ICH have been the first team to identify a novel recessive mutation in the transcriptional regulator LHX4 in a family with severe hypopituitarism – a condition that describes the loss of all pituitary hormones.

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)

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.