Children and young people

NHS England responds to the publication of the independent review of gender identity services for children and young people

An NHS spokesperson said: “NHS England is very grateful to Dr Cass and her team for their comprehensive work on this important review over the past four years. “The NHS has made significant progress towards establishing a fundamentally different gender service for children and young people – in line with earlier advice by Dr Cass […]

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First-ever therapy for rare genetic disease in babies to save lives on the NHS

Babies and toddlers with a rare and fatal genetic condition can now receive a life-saving treatment on the NHS for the first time. Sebelipase alfa (Kanuma®), an enzyme replacement therapy, will become the first treatment available on the NHS for Wolman disease, a condition which presents in babies and children under two years old. Following […]

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New NHS treatments helping extend survival for babies with rare muscle-wasting disease

Nearly three in four babies born with a rare muscle-wasting disease are now surviving for two years or more thanks to advances in NHS treatment, new data shows. Around 70 children are born with spinal muscular atrophy (SMA) each year in the UK, a rare genetic condition that causes muscle weakness, progressive loss of movement […]

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