Apitegromab phase 3: myostatin-blocker improves movement in nonambulatory SMA

Clinical 95/100Rated 95/100Source typePeer-reviewed studyIndependent sourcesstill being verifiedEvery claim links to the page it came from, and stronger sources rate higher. Full methodology →
September 30, 2025↗ source

The SAPPHIRE trial (Lancet Neurology, September 2025) tested apitegromab - a myostatin-targeting antibody - atop existing SMA therapy in 240 nonambulatory children and adults. It met its primary endpoint with meaningful motor-function improvement versus placebo, the first phase 3 win for the myostatin-blocking approach in neuromuscular disease.

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