Showing 118 results
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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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Press release /SMART study to extend data beyond patient population studied in clinical trials New clinical study to evaluate safety and efficacy of Zolgensma in children up to 21 kg, adding to real-world…
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Press release /Children with SMA treated presymptomatically achieved age-appropriate motor milestones including sitting, standing and walking; required no ventilatory or feeding tube support; and had no serious,…
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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…