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Early gene therapy stops spinal muscular atrophy in babies – Medicine

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Early gene therapy stops spinal muscular atrophy in babies – Medicine

(ANSA) – ROME, MARCH 18 – Gene therapy works on young patients with spinal muscular atrophy (SMA): children treated early and before the onset of symptoms ‘grow like healthy peers’, developing the skills expected of children of that age. It is, according to a Novartis note, the success of ‘onasemnogene abeparvovec’, the first and only gene therapy for SMA that is given once in a lifetime.

The phase III SPR1NT study demonstrated that presymptomatically treated children achieved age-appropriate motor milestones, including the ability to stand and walk. In addition, an analysis of three other clinical trials indicated that, following treatment with onasemnogene abeparvovec, children with SMA type 1 achieved or maintained important milestones, including the ability to speak, swallow, meet nutritional needs and maintain airway protection. The findings were presented at the Muscular Dystrophy Association (MDA) 2022 Clinical and Scientific Conference. Onasemnogene abeparvovec is the only SMA treatment designed to directly address the genetic cause of the disease by replacing the function of the missing or non-functioning SMN1 gene in order to stop disease progression with a single intravenous administration. Onasemnogene abeparvovec is approved in over 40 countries, and more than 1,800 patients have now been treated globally. “The results of the SPR1NT clinical trial once again confirm the significant impact of onasemnogene abeparvovec on children who are treated before symptoms appear. In stark contrast to the natural course of the disease, children who are treated preventively remain standing and walking, with few or no signs of neuromuscular disease. Many of these children achieve motor development patterns indistinguishable from their healthy peers, “says Kevin Strauss, Medical Director of the Clinic for Special Children in Pennsylvania. “These data clearly demonstrate the value of neonatal screening for SMA, which is critical for earlier diagnosis and treatment.” (HANDLE).

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