FDA Approves AveXis’ Zolgensma for Treatment of Spinal Muscular Atrophy in Pediatric Patients

Today, the U.S. Food and Drug Administration (FDA) approved Zolgensma (onasemnogene abeparvovac-xioi), the first gene therapy for a neuromuscular disease. Zolgensma is a one-time intravenous (into the vein) infusion for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene, . . .

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2019 MDA Engage ALS Symposium Scheduled in Boston on May 18

At MDA, we believe in the power of research and the importance of building relationships among families, clinicians, and the scientists making discoveries to advance treatments and therapies. We invite individuals living with a neuromuscular disease, as well as caregivers and loved ones, to attend MDA Engage symposia taking place across the country. MDA Engage . . .

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Muscular Dystrophy Association Bridges Clinical and Academic Neuromuscular Research for the First Time at Annual Meeting

With a laser focus on new horizons in neuromuscular research and care, the Muscular Dystrophy Association (MDA) is combining its clinical and scientific conferences for the first time, establishing a robust, state-of-the-art gathering for both clinicians and investigators from across the nation. Medical and scientific experts in neuromuscular disease from academia, private practice, government, and . . .

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FDA Accepts AveXis’ BLA for SMA Drug Zolgensma Under Priority Review

Today, Novartis, the parent company of AveXis Inc., announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) under Priority Review for Zolgensma (formerly known as AVXS-101), a gene therapy for the treatment of spinal muscular atrophy (SMA) type 1. Priority Review status requires the FDA to review . . .

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