Great Ormond Street Hospital (GOSH) has been announced as the lead in a new Europe-wide epilepsy network, aiming to increase collaborations across Europe and improve access to innovative and highly-specialised diagnostics.
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 wit
Research carried out by BRC-funded Professor Paul Brogan and his team has defined a new autoinflammatory disease (AID) in humans with periodic fevers, immunodeficiency and intermittent thrombocytopenia.