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Great Ormond Street Hospital Conference 2017 – ‘Advances in Paediatrics’


We are firm believers that the amazing work of our staff needs to be recognised. The staff at Great Ormond Street Hospital (GOSH) produce research at many different levels ranging from excellent clinical audits to randomised control trials.

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

A new protein that appears to play a role in mitochondrial disease – a rare condition where a lack of energy in cells means that they can’t function properly – could prove to be important in conditions such as Alzheimer’s, Parkinson’s and Huntington’s diseases.

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.

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.

Advances in the treatment of the severe immune disorder Wiskott-Aldrich syndrome

Researchers at Great Ormond Street Hospital (GOSH) have made an important breakthrough in understanding how the immune system becomes disrupted in Wiskott-Aldrich syndrome (WAS), a genetic disorder where the patient’s immune cells begin attacking their own body.