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Biomedical Research Centre news

Advances in the treatment of the severe immune disorder Wiskott-Aldrich syndrome

20 Nov 2017, 2:29 p.m.

Researchers at Great Ormond Street Hospital (GOSH) have made an important breakthrough in understanding how the immune system becomes disrupted in Wiskott-Aldrich syndrome (WAS), a genetic disorder where the patient’s immune cells begin attacking their ow

Drug for Duchenne Muscular Dystrophy shows promise in clinical trial

21 Aug 2017, 12:02 p.m.

An international clinical trial has found that the drug ataluren can improve mobility for children with a severe form of Duchenne muscular dystrophy (DMD).

£75,000 boost for Duchenne Muscular Dystrophy gene therapy research

8 Aug 2017, 4:03 p.m.

GOSH Biomedical Research Centre researcher Dr John Counsell has been awarded up to £75,000 of funding from the UCL Therapeutic Acceleration Support (TAS) Fund for a study exploring the use of novel vectors in gene therapy for Duchenne Muscular Dystrophy (

GOSH researchers receive UCL Business Award

23 Jun 2017, 12:57 p.m.

Professors Bobby Gasper and Adrian Thrasher, both members of the BRC senior management team, have been awarded the UCL Business Award, in recognition of their work on the spin-out company Orchard Therapeutics.

Cannabidiol trial shows reduction in seizures for children with Dravet syndrome

30 May 2017, 12:58 p.m.

A trial conducted in Europe and the USA has shown that cannabidiol – a drug derived from cannabis but with the psycho-active elements removed – reduces seizures in children with a form of drug resistant epilepsy, known as Dravet syndrome.