Apitegromab Over 1 Year Bolsters Motor Abilities of Types 2 and 3: TOPAZ Trial

Apitegromab Over 1 Year Bolsters Motor Abilities of Types 2 and 3: TOPAZ Trial

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Apitegromab Over 1 Year Bolsters Motor Abilities of Types 2 and 3: TOPAZ Trial

Apitegromab, Scholar Rock’s muscle-directed therapy for spinal muscular atrophy (SMA), safely and effectively improved or stabilized motor function in children and young adults with types 2 and 3 disease over one year, top-line data from the Phase 2 TOPAZ trial show. Notably, patients’ motor gains were either maintained or increased at one year relative to what was observed at six months, highlighting the durability and continuous therapeutic potential of apitegromab. “These top-line 12-month data provide further support towards establishing…

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