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Biomedical Research Centre news

Burosumab treatment for XLH Rickets_before and after

Precision medicine improves growth for children with a rare form of rickets

6 Jul 2018, 10:39 a.m.

A new, targeted drug has been shown to significantly improve growth and control of rickets in children with X-linked hypophosphataemic rickets (XLH), a serious condition affecting about 300 children in the UK.

Meredyth Wilkinson, one of the study's first authors

Cutting edge new method reveals novel cause of Juvenile dermatomyositis

25 Jun 2018, 10:18 a.m.

A new in-depth method has helped shed light on the key role of B cells, a type of immune cell, in the rare childhood condition Juvenile dermatomyositis (JDM).

Novel therapy reduces disease progression in rare neurodegenerative condition

21 Jun 2018, 11:55 a.m.

A pioneering new treatment trialled at Great Ormond Street Hospital (GOSH) reduces disease progression by 80 per cent for patients with the rare neurological disorder CLN2.

Cannabidiol reduces seizures for children with complex epilepsies

14 Jun 2018, 8:49 p.m.

Cannabidiol, a drug derived from cannabis but with the psychoactive ingredients removed, has been shown to reduce seizures by around 40 % in children with a rare form of drug resistant epilepsy known as Lennox-Gastaut syndrome (LGS).

£1 million award to develop gene therapy for rare blood disorder FHL-2

12 Jun 2018, 9:31 a.m.

Professor Bobby Gaspar is to receive investment of up to £1million from the UCL Technology Fund (UCLTF) to support development of a gene therapy for familial haemophagocytic lymphohistiocytosis (FHL type 2 or ‘FHL-2’) through to a clinical trial.