Showing 123 results
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Press release /Children with three copies of SMN2 treated presymptomatically achieved age-appropriate milestones, including standing and walking; required no ventilatory or feeding tube support; and had no serious…
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Press release /All children (100%) treated presymptomatically in the SPR1NT two-copy cohort survived without respiratory or nutritional support, and sat independently for ≥30 seconds, most (11/14) within the WHO…
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Press release /The SMART study supplements a growing body of evidence on the use of Zolgensma in a patient population older and heavier (1.5 – 9.1 years of age) than the children treated in previous clinical…
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Clinical Trials /
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Press release /The FDA's May 2019 approval of Zolgensma ®, a gene therapy for spinal muscular atrophy (SMA) in pediatric patients less than 2 years of age, marked an important milestone within the SMA community.…
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Ad hoc release /One-time treatment with Zolgensma (onasemnogene abeparvovec-xioi) is designed to replace lifetime of chronic therapy for all pediatric patients with SMA Annualized cost of Zolgensma is USD…
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Press release /Ph 3 STR1VE data show prolonged event-free survival, early and rapid increases in CHOP-INTEND and significant milestone achievement in SMA Type 1, consistent with START trial First-in-human…
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Ad hoc release /Zolgensma® (onasemnogene abeparvovec), a one-time administered gene therapy, has been recommended for European Commission (EC) conditional approval for patients with spinal muscular atrophy (SMA) and…
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Press release /Interim long-term follow-up data showed all enrolled Cohort 2 patients maintained motor function and milestones achieved during the Phase 1 START trial Mean age of follow-up since dosing…