spinal muscular atrophy

Apitegromab (SAPPHIRE): phase 3 trial shows modest motor gains in non‑ambulatory type 2/3 spinal muscular atrophy on SMN therapies

By MedXY 08/22/2025

The SAPPHIRE phase 3 trial found that adding apitegromab to nusinersen or risdiplam produced a small but statistically significant improvement in motor function in children aged 2–12, with a safety profile similar to placebo.