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1. Spinal muscular atrophy diagnosis in latin american: the registrame clinical registry

6. P.03 Myosin dysregulation in nemaline myopathy

12. SMA – THERAPY: P.271 FIREFISH Parts 1 and 2: 12-month pooled safety and efficacy outcomes of risdiplam (RG7916) in infants with Type 1 spinal muscular atrophy (SMA)

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