Overview
- NICE issued final draft guidance recommending nusinersen and risdiplam for routine commissioning in England after years of time‑limited access.
- NHS England said a new commercial deal guarantees lifelong treatment for eligible patients and confirmed the one‑shot gene therapy Zolgensma is also routinely commissioned.
- National SMA REACH UK data reported 73 children with the most severe form have now reached age five or older on therapy, reflecting improved survival and function.
- The agreement includes a newly approved reduced‑dose nusinersen regimen that halves initial injections and then shifts to one dose every four months.
- An Oxford‑led, NIHR‑funded study will screen about 750,000 newborns in phases to test adding SMA to the heel‑prick check, a push boosted by Jesy Nelson’s advocacy after her twins’ diagnoses.