FDA Approves First Gene Therapy for Spinal Muscular Atrophy

TUESDAY, May 28, 2019 — The first gene therapy has been approved to treat children younger than 2 years with spinal muscular atrophy (SMA), the U.S. Food and Drug Administration announced Friday.
Zolgensma (onasemnogene abeparvovec-xioi), an…
Source: Topamax