The Medical Letter on Drugs and Therapeutics
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ISSUE
1577
July 29, 2019
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Issue 1577
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Zolgensma - One-Time Gene Therapy for Spinal Muscular Atrophy
July 29, 2019 (Issue: 1577)
The FDA has approved onasemnogene abeparvovec-xioi (Zolgensma – Avexis), an adeno-associated virus vector-based gene therapy, for one-time IV treatment of children <2 years old who have spinal muscular atrophy (SMA) and bi-allelic mutations...more
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