ANCORA IMPARO: WHAT'S HAPPENING. MDA CELEBRATES FDA APPROVAL OF ZOLGENSMA FOR TREATMENT OF SPINAL MUSCULAR ATROPHY.

The article reports on a celebration held by the Muscular Dystrophy Asssociation (MDA) following the drug approval given by the U.S. Food and Drug Administration (FDA) to Zolgensma, the first gene therapy for a neuromuscular disease. The drug is designed to target the genetic root cause of spinal muscular atrophy (SMA) by delivering the survival motor neuron gene. Comment from MDA president and chief executive officer Lynn O'Connor Vos about the breakthrough is presented.