Professors Graham Davies and Adrian Thrasher have been awarded funding from Great Ormond Street Hospital (GOSH) Charity to further develop a cutting-edge new treatment for children with the rare immunodeficiency condition complete DiGeorge syndrome (cDGS)
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.
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).