Medicines watchdog approves two treatments for patients with spinal muscular atrophy
Hundreds 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The National Institute for Health and Care Excellence (Nice) has published final draft guidance recommending that any patient who would benefit can have either drug.
The move means that anyone in England, Wales or Northern Ireland with spinal muscular atrophy will from Thursday be able to get either nusinersen, also known as Spinraza, or risdiplam, also known as Evrysdi, from the NHS.
SMA is a progressive genetic disorder that causes severe muscle weakness and can affect the ability to move, breathe and swallow. Without treatment, patients face devastating consequences including profound disability and reduced life expectancy. Children with the most severe form of SMA – type 1 – usually die before they reach two.
