In a first-of-its-kind study, researchers compared the efficacy of preventative therapy for spinal muscular atrophy (SMA) between two well-matched study groups, using either gene therapy (onasemnogene abeparvovec) alone or in combination with risdiplam (oral medication) or nusinersen (intrathecal injection) administered before apparent signs of...
Researchers evaluate the benefit of dual therapy for children at risk for spinal muscular atrophy retrieved 25 June 2024 from https://medicalxpress.com/news/2024-06-benefit-dual-therapy-children-spinal.html
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