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Charlie and Rupert: Spinal Muscular Atrophy and Zolgensma

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Spinal Muscular Atrophy

Charlie and Rupert on their journey with Spinal Muscular Atrophy and Zolgensma gene therapy.

Rupert was diagnosed with Spinal Muscular Atrophy type 1 when he was 8 weeks old. He received the gene therapy Zolgensma, an advanced therapy medicinal product (ATMP), at 11 weeks old.

Charlie tells the NHS Accelerated Access Collaborative about her journey to getting a diagnosis for Rupert, the implications on her family, the support she received and her experience as Rupert received a novel gene therapy.

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