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Treatment Evolution in 5q-Spinal Muscular Atrophy: Real-World Evidence and Clinical Insights from the SMArtCARE Registry
Posted inClinical Updates Neurology news

Treatment Evolution in 5q-Spinal Muscular Atrophy: Real-World Evidence and Clinical Insights from the SMArtCARE Registry

Posted by MedXY By MedXY 03/08/2026
This review analyzes the shifting landscape of SMA therapy using data from 2,140 patients, highlighting the transition between nusinersen, risdiplam, and onasemnogene abeparvovec and the factors driving treatment switches.
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Apitegromab (SAPPHIRE): phase 3 trial shows modest motor gains in non‑ambulatory type 2/3 spinal muscular atrophy on SMN therapies
Posted inNeurology Specialties

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

Posted by MedXY 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.
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