Life-saving drug approved for use

Children with the most severe form of a genetic condition will now have access to a “lifechanging” treatment after it was approved for use by the NHS in Scotland.

Muscular Dystrophy UK says that Spinraza is the first and only treatment for patients with spinal muscular atrophy (SMA), a rare and inherited muscle-wasting condition that can cause patients to lose the ability to move, breathe and swallow.

Without access to it, life expectancy for those with SMA type one is rarely longer than two years.

The Scottish Medicines Consortium (SMC) recommended yesterday that the drug be made available on the NHS in Scotland for children with type one SMA. It did not, however, approve the treatment for patients with type two and three of the condition.

Muscular Dystrophy UK, which has been campaigning for access to Spinraza for all patients with the condition, welcomed the news but said it will keep fighting until all patients with SMA have access to the treatment.

The drug is one of five approved yesterday, including Regorafenib (Stivarga) which treats liver cancer in patients who have had other treatment.