A life-changing drug that can improve mobility in children and adults with a rare genetic condition will be available on the NHS following a new commercial deal.

NHS England chief executive Amanda Pritchard announced that Risdiplam will be used to treat hundreds of patients a year with Spinal Muscular Atrophy, a rare and often fatal genetic disease that causes paralysis, muscle weakness and progressive loss of movement.

The drug, the first non-injectable treatment for SMA that can be taken at home, improves motor function and is taken as a syrup medicine once a day after meals. At least 215 people in England have already had Risdiplam via the early access scheme provided earlier this year.

This is the third treatment for SMA secured by the NHS in less than three years, transforming care of patients with the condition and means there is now an innovative drug available for individuals with SMA types 1, 2 and 3.

Pritchard said: “In the last three years the NHS has revolutionised care for people with Spinal Muscular Atrophy, by securing access to a trio of innovative treatments- Spinraza, Zolgensma and now Risdiplam- whereas three years ago clinicians had no effective medicines at all.

“SMA is a cruel disease and the leading genetic cause of death among babies and young children, which is why NHS England has been determined to make these treatment available to people as soon as possible to help transform the lives of patients and their families.

“It is yet another example of the NHS leading the way in securing access to the newest and most innovative treatments and therapies available for patients, not only for those with rare genetic conditions, but in all areas including fighting cancer and Covid19 in line with the NHS Long Term Plan.”