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 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).
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 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.
Researchers at GOSH and ICH have been the first team to identify a novel recessive mutation in the transcriptional regulator LHX4 in a family with severe hypopituitarism – a condition that describes the loss of all pituitary hormones.
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.
All hospitals are required to publish information about the numbers of nurses working on each ward day and night, both registered and non registered, together with the percentage of shifts meeting safe staffing guidelines.
The Department of Endocrinology and Diabetes at Great Ormond Street Hospital (GOSH), in partnership with adolescent and adult services at University College London Hospitals (UCLH), form the London Centre for Paediatric Endocrinology and Diabetes.
Steroids are hormonal chemical messengers that are produced naturally in the body by the adrenal glands (which are just above each kidney) and by the reproductive organs. Man-made versions of these hormonal substances are used to treat a wide range of illnesses and medical conditions.