The NIHR Great Ormond Street Biomedical Research Centre (BRC) has been awarded £37 million in funding to drive forward translational research into rare diseases in children. The Centre is the only one of its kind in the UK dedicated to paediatric research.
The prospect of widespread access to a life-changing drug for children with a rare muscular disorder is a step closer today after the United States Food and Drug Administration (FDA) granted accelerated approval for a new medication.
A team led by Professor Paolo De Coppi has announced that they are set to build and transplant an oesophagususing organs harvested from pigs and then modified using a child’s stem cells.The organs will be created for children who are born with a severe cases of oesophageal atresia, where their oesophagus has not formed properly.
Researchers – led by Great Ormond Street BRC-supported Professor Tessa Crompton in collaboration with the Paediatric Department at Oxford University – have identified the role of a key protein in normal development of the thymus, an important organ of the immune system.
Professors Bobby Gasper and Adrian Thrasher, both members of the BRC senior management team, have been awarded the UCL Business Award, in recognition of their work on the spin-out company Orchard Therapeutics.
A trial conducted in Europe and the USA has shown that cannabidiol – a drug derived from cannabis but with the psycho-active elements removed – reduces seizures in children with a form of drug resistant epilepsy, known as Dravet syndrome.
Research led by Diagnostics and Imaging Theme lead, Professor Neil Sebire aimed to investigate which aspects of post-mortem examinations in stillbirths are most effective at providing a cause of death.
A drug for Duchenne Muscular Dystrophy (DMD), originally developed by BRC Theme Lead Professor Francesco Muntoni’s Consortium in the UK, has been filed by Sarepta Therapeutics for accelerated approval by the United States Food and Drug Adminstration (FDA).
Diagnostics and Imaging Theme Lead Professor Neil Sebire has teamed up with researchers from nine other establishments to carry out the largest randomised trial to investigate the long-term safety of vaginal progesterone prophylaxis for preterm births.
Friday 12 May is International Nurses Day and across the world, the global health family comes together to celebrate the nursing profession. This year the theme focuses on ‘nurses as heroes’ and here at Great Ormond Street Hospital (GOSH) we want to pay tribute and say thank you to all our nursing staff who we see as the superheroes of healthcare.
Professor Neil Sebire – who is Diagnostics and Imaging Theme lead at the Great Ormond Street BRC has been awarded an NIHR HTA grant on behalf of the Minimally Invasive Autopsy team at Great Ormond Street Hospital.
At Great Ormond Street Hospital (GOSH), we try to achieve the highest standards in our clinical care and also in the services we provide for children, young people and families. We want to exceed your expectations so we try to improve what we do and how we do it all the time.