Insight

SMA: The new life-changing drug that can be taken at home for patients with rare genetic condition

ITV News Health Editor Emily Morgan hears from two sisters who say the new drug is slowly changing their lives


It doesn’t seem a minute ago since we reported on NICE approving the use of the world’s most expensive drug, Zolgensma, in England.

That’s because it was just a minute ago, it was only in March, and by June the first tiny patient with Spinal Muscular Atrophy (SMA) received the treatment. Now, five months later we’re reporting on another drug approved for SMA patients.

Only this time, Risdiplam can be used at home and is nowhere near as expensive.

This is so exciting because SMA is the leading genetic cause of death among babies and young children and because it can significantly slow the progress down of this brutal neuromuscular condition.


11-year-old Shaya Sfalsin says she can pick up more heavy objects thanks to the medicine


There are now three treatments available for patients on the NHS.

Two have to be administered in hospital, this new one is a syrup and taken at home.

The head of the NHS, Amanda Pritchard, said they have revolutionised care for people with SMA and she is right. Patients now have choices and hope that they can live as normal a life as possible for as long as possible.


Hamane Sfalsin, 14, says even her PE teachers have noticed a difference in her physical ability since taking the new SMA medicine


There are three types of SMA.

Type 1 sadly is the most severe and most children don’t live beyond the age of two, those with type 2 and 3 can live very long lives, though the condition can cause paralysis, muscle weakness and loss of mobility.

Anything any drug can do, to slow the progression of this disease down, is a bonus. Access to treatment early is also absolutely essential, because the earlier children are treated the better results they have. It is always exciting reporting on new, revolutionary medicines. It is even more exciting when you know it’ll help one and a half thousand, many of them children, live a better, life.