Specialised commissioning

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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NHS patients with rare genetic disorders to be fast-tracked to earlier diagnosis and specialist care

Hundreds of people with a range of rare conditions primarily affecting the central nervous system will be fast-tracked to diagnosis and specialist care, thanks to the launch of a pioneering new NHS service. Patients with inherited white matter disorders (IWMDs), also known as leukodystrophies, will now have rapid access to expert teams, increased virtual support […]

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NHS to offer licensed cannabis-based medicine to treat rare genetic condition

The NHS will be able to prescribe cannabidiol to patients with a rare, seizure-causing genetic disorder in England from today (Tuesday) after the National Institute for Health and Care Excellence (NICE) issued final guidance recommending its use. Around one in every 6,000 people suffer from tuberous sclerosis complex (TSC) that causes seizures, which severely affect […]

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NHS treats first patient with the ‘world’s most expensive drug’

A five-month old baby has become the first patient to receive a potentially life-saving drug on the NHS that can prevent paralysis and prolong the lives of children with Spinal Muscular Atrophy (SMA). Arthur Morgan, who was diagnosed with SMA earlier this month, received the one-off gene therapy at Evelina London Children’s Hospital on May […]

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NHS England strikes deal on life-saving gene-therapy drug that can help babies with rare genetic disease move and walk

A life-saving drug that can enable mobility in babies and young children suffering from a rare genetic condition will be available on the NHS, chief executive Sir Simon Stevens announced today. Zolgensma, which has a reported list price of £1.79 million per dose and is labelled the most expensive drug in the world, will be […]

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