A new test to help diagnose and predict a range of serious childhood eye conditions has been developed by researchers at Great Ormond Street Hospital (GOSH) and the UCL Great Ormond Street Institute of Child Health (ICH).
Levels of myostatin, a protein that prevents muscle growth, could influence how well the body responds to anti-myostatin treatments for muscle-wasting conditions such as Duchenne Muscular Dystrophy (DMD). This breakthrough could help predict which patients will benefit from therapies that aim to increase muscle strength by blocking myostatin.
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 own body.
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 (DMD).
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.