NHS to roll out 'life-changing' treatments for children with rare muscle-wasting condition
NICE says nusinersen and risdiplam can improve survival and independence for people with spinal muscular atrophy.
- The National Institute for Health and Care Excellence approved nusinersen and risdiplam for routine NHS use in England, securing long-term access to these treatments for patients with spinal muscular atrophy .
- Previously available through a special access programme, these therapies address the most severe SMA Type 1, which without treatment limits life expectancy to two years in affected infants.
- Nine-Year-Old Ezra Thorman from Ramsgate, Kent, began nusinersen treatment at five months old following his 2016 diagnosis; NHS England data shows the therapies helped 73 children with severe SMA Type 1 survive to age five or older.
- Alongside this approval, scientists at the University of Oxford will screen approximately 750,000 newborns in England to determine whether SMA should be added to routine post-birth health checks.
- Pop star Jesy Nelson and charities campaigned for wider screening access, with Giles Lomax, chief executive of SMA, calling the approval a "historic moment" as Public health minister Sharon Hodgson noted the decision enables fuller, more independent lives.
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'Life-changing' SMA treatments approved for routine NHS use offer hope to thousands
Two groundbreaking treatments for the muscle-wasting condition, spinal muscular atrophy (MSA), have secured approval for routine NHS use in England, offering hope to hundreds of children affected by this rare genetic disorder.The National Institute for Health and Care Excellence has given the green light for nusinersen and risdiplam to be made widely available, marking a significant milestone for families who have long campaigned for permanent a…
The Independent
The Independent (US)‘Life-changing’ SMA treatments to be made routinely available on NHS
Nelson has campaigned for screening to be rolled out after her twins were diagnosed with the condition
NHS To Roll Out 'life-changing' Spinal Muscular Atrophy Treatments For Children
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 fro…
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