Apitegromab Evidence: Trial Results and Peer-Reviewed Publications

Hipa.ai Research · Source: PubMed & ClinicalTrials.gov / AACT · Last updated: June 2, 2026

The clinical evidence base for Apitegromab comprises 1 peer-reviewed publications across 1 journals, 0 pivotal-trial primary-outcome rows reported to ClinicalTrials.gov, spanning indications including Muscular Atrophy, Spinal. Most recent publication: 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.

Top peer-reviewed publications

Curated set of pivotal-trial result papers and recent publications in high-tier journals.

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. · Lancet Neurol · 2025
PubMed: PMID 40818473 · NCT05156320 (SAPPHIRE) · Muscular Atrophy, Spinal

Publications by year

2025–2025: 1 publications.

2025

Publications by indication

Muscular Atrophy, Spinal (1)

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 · PMID 40818473 · NCT05156320

Publications by journal

Lancet Neurol1 paper

All Apitegromab publications (1)

2025 (1 paper)

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. · Lancet Neurol · 2025 · Derived
PubMed: PMID 40818473 · NCT05156320 (SAPPHIRE) · Muscular Atrophy, Spinal

Sources and methodology

Publications: ctgov.study_references (PubMed PMIDs auto-attached by ClinicalTrials.gov to each trial), reference types RESULT, DERIVED, and BACKGROUND.
Per-arm outcome values: ctgov.outcome_measurements joined to ctgov.design_outcomes where outcome_type = 'PRIMARY', restricted to phase PHASE3 and PHASE2/PHASE3.
Publication metadata (title, journal, year, authors): PubMed E-utilities efetch.fcgi
Data freshness: Tue, 02 Jun 2026 17:31:08 GMT.

This page summarizes published evidence for general reference and does not constitute medical advice. For clinical decisions, consult the linked primary publications and your healthcare provider. Data sourced from PubMed and the ClinicalTrials.gov / AACT database maintained by the Clinical Trials Transformation Initiative (CTTI).