NHS to roll out 'life-changing' spinal muscular atrophy treatments for children

Hundreds more children with a rare muscle-wasting condition are to be given access to two "life-changing" treatments after they were approved for NHS use.

NHS England will make two treatments for spinal muscular atrophy (SMA) routinely available after approval by the National Institute for Health and Care Excellence (NICE).

A commercial agreement giving lifelong access to the medication on the NHS to all eligible patients has also been reached.

Nusinersen is given as a regular injection into spinal fluid, while risdiplam is provided as a daily syrup or tablet that can be taken at home.

A study from SMA REACH UK found the medications helped 73 children with the severe form of SMA (Type 1) survive to age five or older – more than half of those treated.

Professor James Palmer, national medical director for specialised services at NHS England, said: "These lifeline treatments have offered a phenomenal step forward in care for children and families affected by such a debilitating condition.

"For parents who faced the unimaginable pain of thinking their child would not reach their second birthday, they now have hope of seeing them walk to school and play with their friends."

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SMA is a rare genetic condition that causes progressive muscle weakness and loss of movement and can affect a child's ability to breathe and swallow.

It's estimated that around 70 babies are born with the condition each year in the UK, with SMA often grouped into types based on the age at which symptoms begin and how it affects sitting, standing and walking.

According to the NHS, around 1,150 people in England are living with SMA types one, two, or three.

It's been brought to national attention in part by singer and former Little Mix star Jesy Nelson, whose twins, Ocean Jade and Story Monroe Nelson, were diagnosed with the condition.

Speaking to Sky News' The UK Tonight With Sarah-Jane Mee in January, Nelson said that "it took for my mum to spot that sign and that's what's really worrying".

Helen Knight, director of medicines evaluation at NICE, said: "After carefully reviewing the latest evidence and real-world experience from the NHS, our independent committee concluded that nusinersen and risdiplam can offer substantial, life-changing benefits for many people with SMA.

"These treatments can help people live longer, maintain their independence, communication and participation in everyday life, and reduce the need for hospital care."

(c) Sky News 2026: NHS to roll out 'life-changing' spinal muscular atrophy treatments for children