Child at Royal Manchester Children’s Hospital is first in north to receive £1.79m life-saving gene-therapy drug
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manchestereveningnews.co.ukDon't miss a thing by getting the day's biggest stories sent direct to your inbox A patient at Royal Manchester Children’s Hospital is the first in the north of England to receive a potentially life-saving drug on the NHS .
Zolgensma is a one-off gene therapy that treats Spinal Muscular Atrophy (SMA) - a rare and often fatal genetic disease that causes paralysis, muscle weakness and progressive loss of movement.
It has a list price of £1.79 million per dose, but was made available on the NHS following a landmark deal struck with manufacturers Novartis Gene Therapies, back in March.
The latest data suggests that Zolgensma can provide improvement in motor function for young children with a specific type of SMA and prolong their lives.
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