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.
XLH is caused by genetic mutation that leads to excess activity of fibroblast...
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.
Giving the drug sodium thiosulphate after chemotherapy reduces hearing loss in children treated for liver cancer, according to findings published in the New England Journal of Medicine today (Wednesday).
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).
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.
A whole team of people carry out research in Great Ormond Street Hospital. To thank them all on this International Clinical Trials Day, we’re sharing a picture drawn by one of the patients being seen.
Research led by Professor Lyn Chitty has shown that prenatal exome sequencing in fetuses can provide accurate and timely molecular diagnoses for skeletal conditions and inform pregnancy management.
