NHS drugs go-ahead offers lifeline to children with rare muscle-wasting disease
Key takeaways
- Ezra Thorman started treatment with nusinersen when he was a baby and his mother, Portia, says it saved his life.
- Prefer the Guardian on GoogleHundreds of children with a rare muscle-wasting disease will be able to receive two drugs that can improve their survival in a move parents hailed as a “lifeline”.
- The National Institute for Health and Care Excellence (Nice) has published final draft guidance recommending that any patient who would benefit can have either drug.
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Ezra Thorman started treatment with nusinersen when he was a baby and his mother, Portia, says it saved his life. Photograph: Courtesy of Thorman family View image in fullscreen Ezra Thorman started treatment with nusinersen when he was a baby and his mother, Portia, says it saved his life. Photograph: Courtesy of Thorman family Children NHS drugs go-ahead offers lifeline to children with rare muscle-wasting disease Medicines watchdog approves two treatments for patients with spinal muscular atrophy
Prefer the Guardian on GoogleHundreds of children with a rare muscle-wasting disease will be able to receive two drugs that can improve their survival in a move parents hailed as a “lifeline”.
The National Institute for Health and Care Excellence (Nice) has published final draft guidance recommending that any patient who would benefit can have either drug.