Families seek access to 'life-changing' drug

Herald Scotland

3 April 2018 - Patients with the most severe form of a genetic condition could lose the ability to move, breath and swallow unless a "life-changing" treatment is approved for use on the NHS in Scotland, according to a charity.

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.

Without access to it, life expectancy for those with SMA type 1 - the most severe form - is rarely longer than two years, campaigners warn, and they claim the drug can also help people with other forms of SMA.

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Michael Wonder

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Michael Wonder