Study examines safety and early outcomes of gene therapy for spinal muscular atrophy

Study examines safety and early outcomes of gene therapy for spinal muscular atrophy

Study examines safety and early outcomes of gene therapy for spinal muscular atrophy

In May 2019, the U.S. Food and Drug Administration approved a gene replacement therapy for the inherited, progressive neuromuscular disease 5q-linked spinal muscular atrophy.