REGISTRE SMA FRANCE: A nationwide observational registry of patients with spinal muscular atrophy in France
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BackgroundSpinal muscular atrophy (SMA) is a severe neurodegenerative disease affecting children. Three innovative disease-modifying therapies (DMTs)-nusinersen, risdiplam, and onasemnogene abeparvovec-are available for treatment.ObjectiveTo provide a descript... ...
