Researchers from the UCL Great Ormond Street Institute of Child Health (ICH) and Great Ormond Street Hospital (GOSH) will lead a UK-wide effort to drive the development of new, targeted treatments for children and young people with juvenile idiopathic art
Researchers at Great Ormond Street Hospital (GOSH) and the UCL Great Ormond Street Institute of Child Health (ICH) are to lead a new collaboration exploring how stem cells can be engineered to evade immune responses.
A new artificially intelligent bodysuit to help understand how mobility is affected in boys with Duchenne will be trialled in a collaboration between researchers at Great Ormond Street Hospital (GOSH) and Imperial College London.
A new collaboration aiming to develop advanced technologies for manufacturing ex vivo gene therapy products has been established between researchers at UCL Great Ormond Street Institute of Child Health (ICH) and Plasticell, a developer of stem cell techno
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).
An international clinical trial has found that the drug ataluren can improve mobility for children with a severe form of Duchenne muscular dystrophy (DMD).
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 retir
Injections of the drug canakinumab significantly reduced the number of fever episodes in children with three different types of recurrent fever conditions, in an international trial led at GOSH by Professor Paul Brogan.
GOSH and UCL Great Ormond Street Institute of Child Health (ICH) researchers Professor Paul Gissen and Dr Julien Baruteau will be part of a new collaboration with Evox Therapeutics that aims to develop a new therapy for the life threatening metabolic cond
A two-year old patient at the Royal Manchester Children’s Hospital (RMCH) is the first in the world to receive a pioneering new gene therapy treatment for the rare and life-limiting genetic condition, Mucopolysaccharidosis IIIA (MPSIIIA). The treatment wa
Last weekend, we opened our doors to over 300 visitors for the annual Family Fun Day hosted by the National Institute for Health Research (NIHR) Great Ormond Street Hospital (GOSH) Biomedical Research Centre (BRC).
GOSH researchers who specialise in childhood brain injury helped shape a new play that tells the story of a little boy astronaut, Jude Starbeam, who gets lost in space.
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).
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.
GSK and Orchard Therapeutics today announced a strategic agreement, under which GSK will transfer its portfolio of approved and investigational rare disease gene therapies to Orchard, securing the continued development of the programmes and access for pat
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 o