Patients with a rare genetic condition that causes progressive muscle weakness are to benefit from a new treatment available on the NHS in England.
Risdiplam boosts the level of a key protein lacking in patients with SMA. This is expected to increase survival of nerve cells that control movement to manage the symptoms of the disease and slow down its progression. Of the three main types of SMA, type 1 is the most serious. Without treatment, children mostly don't survive beyond the age of two.
Risdiplam costs £7,900 per bottle and may last patients just a couple of weeks as dosage depends on weight. "Just to have the certainty that it is not going to get any worse would be huge. I may not get back my ability to walk, that's fine. I'm still happy in the skin that I live in," he said.
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