Children and young people

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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Vision-saving drug to be routinely offered on NHS to prevent blindness in premature babies

Premature babies are to be routinely offered a “life-changing” treatment to prevent blindness on the NHS for the first time, the head of the health service has announced today. The drug, ranibizumab, could save the eyesight of babies born with a condition known as retinopathy of prematurity (ROP), which affects blood vessels in the retina, […]

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