NHS drugs go-ahead offers lifeline to children with rare muscle-wasting disease
Summary
The UK's National Health Service (NHS) will provide two drugs, nusinersen and risdiplam, to treat children with spinal muscular atrophy (SMA), a rare and serious muscle-wasting disease. This decision means patients in England, Wales, and Northern Ireland can access these medications to improve survival and quality of life.Key Facts
- Spinal muscular atrophy (SMA) is a genetic disease causing muscle weakness, difficulty moving, breathing, and swallowing.
- Without treatment, SMA type 1 often leads to death before age two.
- NHS England will offer the drugs nusinersen (Spinraza) and risdiplam (Evrysdi) to all eligible patients.
- Nusinersen is given as injections in spinal fluid; risdiplam is taken daily as a syrup or tablet.
- About 1,150 people under 18 have SMA in England; around 70 babies are born with SMA yearly in the UK.
- The drugs have been available in limited use since 2019 (nusinersen) and 2021 (risdiplam).
- The NHS reached a price deal with drug makers Biogen and Roche to offer these treatments widely.
- Newborn screening for SMA is being evaluated to possibly add to routine tests at birth.
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