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​GOSH ranked fourth in North Thames region for clinical trial activity

Great Ormond Street Hospital for Children NHS Foundation Trust (GOSH) has been ranked 4th in the North Thames region for number of active clinical research studies in 2016/17, in figures released today by the National Institute for Health Research (NIHR).

GOSH awarded £37 million funding to carry out pioneering paediatric research

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.

Chancellor of the Exchequer George Osborne visits Great Ormond Street Hospital and meets young patients

This morning, the Chancellor of the Exchequer George Osborne visited Great Ormond Street Hospital (GOSH) to announce a new £800 million boost to biomedical research through the National Institute for Health Research (NIHR).

Research suggests need for long-term follow-up for patients with Kawasaki Disease

Kawasaki disease is a rare condition that causes blood vessels to become inflamed and swollen. It predominantly occurs in children under the age of five. If left untreated, it results in balloon-like swellings (aneurysms) in the coronary vessels of approximately 25% of cases. To date, the cause of this disease remains unknown.

£75,000 boost for Duchenne Muscular Dystrophy gene therapy research

GOSH Biomedical Research Centre researcher Dr John Counsell has been awarded up to £75,000 of funding from the UCL Therapeutic Acceleration Support (TAS) Fund for a study exploring the use of novel vectors in gene therapy for Duchenne Muscular Dystrophy (DMD).

Researchers announce oesophagus regeneration trial

A team led by Professor Paolo De Coppi has announced that they are set to build and transplant an oesophagus using 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 awarded up to £1 million to develop gene therapy for serious immunodeficiency

The UCL Technology Fund (UCLTF) has approved an investment of up to £1 million to support researchers Professor Adrian Thrasher, Professor Bobby Gaspar, and Dr GiorgiaSantilli in developing a gene therapy approach for treating p47phox-deficient chronic granulomatous disease (p47-CGD).

Identification of genetic mutation linked to complications associated with common birthmarks

Research led by BRC-supported Dr Veronica Kinsler has found that a subset of a common type of birthmark, which is associated with severe complications, is caused by activating mutations in the genes GNAII and GNAQ. These findings could lead to early identification of infants at risk of serious complications.

A new, genetic diagnostic service for ciliopathy disorders has been launched

In a collaboration between Great Ormond Street Hospital (GOSH) North-East Thames Regional Genetics and the UCL Institute of Child Health, a new genetic diagnostic service for ciliopathy disorders has been launched for service delivery to the NHS.

Sir Michael Rake appointed Chairman of Great Ormond Street Hospital for Children NHS Foundation Trust

Sir Michael Rake, currently Chairman of the BT Group plc, has been appointed as the new Chairman of Great Ormond Street Hospital for Children NHS Foundation Trust and will take up the position in November 2017.

Spinraza: the first treatment for spinal muscular atrophy to receive FDA approval

At the end of 2016, the American Food and Drug Administration (FDA) granted approval for the use of Spinraza (nusinsersen) for the treatment of spinal muscular atrophy (SMA) in paediatric and adult patients. This is unprecedented for the SMA community witnessing the first approved drug for this disease.

Protein responsible for rare childhood disorder gives clues to common neurodegenerative conditions

A new mutation in the protein STAT2 has been identified in patients with mitochondrial disease. These findings could also be beneficial for more common neurodegenerative diseases, including Alzheimer’s, Huntington’s and Parkinson’s diseases.

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