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Amedeo Smart

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Crawford TO, Servais L, Mercuri E, Kolbel H, et al. Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial. Lancet Neurol 2025;24:727-739.
PMID: 40818473


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