PubMed · 2025

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

Crawford TO, Servais L, Mercuri E, et al. (2025). 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.

Lancet Neurol

Source: https://pubmed.ncbi.nlm.nih.gov/40818473/

Last reviewed September 19, 2026. By the PeptIQ team.

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