Our brand new medical facility, the Zayed Centre for Research into Rare Disease in Children, brings together pioneering research and clinical care under one roof to drive forward new treatments and cures for seriously ill children, both across the UK and internationally. One of the first patients to be treated at the Centre is Kai, aged 10.
NHS patients who have taken part in clinical trials have expressed their gratitude to researchers in a film celebrating the work of the National Institute for Health Research (NIHR) Biomedical Research Centres (BRCs), including GOSH BRC, to mark the retirement of outgoing Chief Medical Officer Professor Dame Sally Davies.
Rue, 17, has Neuromyelitis Optica, a rare autoimmune condition affecting 1 in 5 million people. It can be characterised by relapses (attacks) of the optic nerve and spinal cord, which can cause vision problems and sight loss. Rue is taking part in a clinical trial to see whether a new drug can help reduce relapses that occur in her vision. Here she shares her story:
Meet physiotherapist Lesley Katchburian who cares for Cora in episode four of Paul O’Grady’s Little Heroes. Lesley, who is Lead Clinical Specialist Physiotherapist for Botulinum Toxin Services in Neurodisability at GOSH, explains why botulinum toxin can be helpful for children with cerebral palsy and why research is so important.
Patients at Great Ormond Street Hospital (GOSH) and across England are set to receive treatment for the rare nervous system condition Battens Disease, following a deal between the NHS and manufacturer Biomarin. The drug, which is called cerliponase alfa or Brineura, has been approved by the NHS following successful clinical trials at GOSH in collaboration with other centres from across the world.
Joe, aged 15, who features on Paul O’Grady’s Little Heroes, is being treated at GOSH for Marfan Syndrome, a genetic disorder that affects the body’s connective tissue, which plays an important role in helping the body grow and develop properly. Joe has an associated heart condition and required surgery to replace his aorta, the body’s main artery.
The medical and research team involved in his care share how they have used pioneering 3D Heart Modelling and Virtual Reality to aid understanding of his heart condition ahead of surgery.
Artificial intelligence (AI) is increasingly being used to improve our everyday lives and, at GOSH we’re proud to be leading the way in applying this sophisticated technology to healthcare research. Dr Valeria Ricotti shares how we've been using AI to understand more about duchenne muscular dystrophy (DMD).
A clinical trial to investigate a novel CAR T-cell therapy, designed to target cancer cells more quickly and cause less side effects, has shown very promising results for children with previously incurable acute lymphoblastic leukaemia (ALL).