Onasemnogene abeparvovec gene therapy for treatment of patients with spinal muscular atrophy: Updated real-world practical considerations
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Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease resulting from biallelic pathogenic variants of the survival motor neuron 1 (SMN1) gene that leads to motor neuron degeneration, progressive muscle atrophy, and weakness. In its most... ...
