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jamaneurology_mendell_2021_oi_210023_1624467431.00495

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Atrofia_muscular_espinal

Gil Lizarraga, Uxue

Spinal Muscular Atrophy

Safety and efficacy of nusinersen in spinal muscular atrophy The EMBRACE study

Spinal muscular atrophy From approved therapies to future therapeutic targets for personalized medicine

New and Developing Therapies in Spinal Muscular

Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy

Five-Year Extension Results of the Phase 1 START Trial

Advances and limitations for the treatment of spinal muscular atrophy