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.
