An international clinical trial has found that the drug ataluren can improve mobility for children with a severe form of Duchenne muscular dystrophy (DMD).
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 (
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.
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.
A collection of 100-year old tumour samples held at Great Ormond Street Hospital (GOSH) has helped pinpoint the genetic mutations that cause some of the rarest childhood cancers.