A new life-changing drug will treat spinal muscular atrophy at home, following a commercial deal made by the NHS

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The NHS has secured a new drug, risdiplam, to treat spinal muscular atrophy (SMA) in children and adults and improve the lives of hundreds of patients a year.

SMA is a rare genetic condition that causes paralysis, muscle weakness, and progressive loss of movement. Risdiplam improves motor function and can be taken at home as a syrup medicine—the first non-injectable treatment for SMA.

The treatment is especially significant for people with SMA where existing treatment options may not be appropriate, such as those who have had spinal surgery.

This is the third treatment for SMA secured by the NHS in less than 3 years; there is now an innovative drug available for people with SMA types 1, 2, and 3. Babies as young as 2 months, with a diagnosis of SMA types 1, 2, or 3, can be treated with risdiplam.

Amanda Pritchard, NHS Chief Executive, 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 COVID-19 in line with the NHS Long Term Plan.’


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