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).
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, 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).
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.
Earlier this month patients and families at GOSH (Great Ormond Street Hospital) were given the chance to get hands-on with science at our Research Activity Trial and with special lessons in the hospital school.