FDA Approves Gene Therapy for Treatment of Spinal Muscular Atrophy
The U.S. Food and Drug Administration approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of spinal muscular atrophy (SMA) in adult and pediatric patients 2 years of age and older with confirmed mutation in the survival motor neuron 1 (SMN1) gene. Itvisma is an adeno-associated virus (AAV) vector-based gene therapy. “Today’s approval shows the power of