Health Professional Radio - Podcast
First Gene Therapy for Spinal Muscular Atrophy
- Autor: Vários
- Narrador: Vários
- Editor: Podcast
- Duración: 0:08:05
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Sinopsis
Dr. Meredith Schultz with AveXis discusses the FDA approval of Zolgensma (onasemnogene abeparvovec-xioi), the first gene therapy for the treatment of spinal muscular atrophy (SMA) in pediatric patients less than 2 yrs of age. It is given as a one-time infusion into the vein. Zolgensma was not evaluated in patients with advanced SMA.
