Oral drug for spinal muscular atrophy to be available on NHS in England

Oral drug for spinal muscular atrophy to be available on NHS in England The NHS is to introduce a revolutionary new treatment to tackle the leading genetic cause of death among babies and young children.

About 1,500 patients in England with certain types of spinal muscular atrophy (SMA) are expected to benefit from risdiplam, after a recommendation from the health watchdog. The drug, also called Evrysdi and made by Roche, is a syrup that can be taken at home and is the first non-injectable treatment for the condition. The Guardian

See also: 

• Risdiplam for treating spinal muscular atrophy in children and adults (draft guideline) NICE
• NICE draft guidance recommends new treatment for spinal muscular atrophy as part of a managed access agreement NICE
• Take at home treatment for spinal muscular atrophy BBC News
• NHS to give life-changing drug to spinal muscular atrophy patients The Independent