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New treatment for a rare genetic disease to be made available on NHS – The indy100

Risdiplam has been recommended for use initially in people with certain types of spinal muscular atrophy.

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A treatment for a rare genetic disorder is being made available on the NHS in the hope it can help transform the lives of children and adults by improving their mobility.
Around 1,500 people in England with certain types of spinal muscular atrophy (SMA) will…

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