Spinal muscular atrophy: Gene therapy approved by NHS
Drug treatment for fatal genetic disorder will be the most expensive approved in the UK. …
Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at Nice, said: “Spinal Muscular Atrophy is a very serious, debilitating and distressing condition that has very significant effects on every aspect of life of those with SMA, and their families and carers.
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