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Efficacy and safety of masitinib in amyotrophic lateral sclerosis patients prior to loss of functionality: A subgroup analysis optimizing the benefit-risk profile of masitinib

2025-12-10 neurology Title + abstract only
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BackgroundAmyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that urgently requires new treatments. Masitinib, a tyrosine kinase inhibitor targeting microglia and mast cells, aims to slow disease progression by reducing neuroinflammation. A previously reported phase 2b/3 study showed that masitinib (4.5 mg/kg/day) with riluzole significantly slowed decline of the revised amyotrophic lateral sclerosis functional rating scale (ALSFRS-R) over 48 weeks in a specific patient populatio...

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