Gene Therapy for Spinal Muscular Atrophy

Adenovirus-mediated gene therapy. National Institutes of Health.

In May 2019, the U.S. Food and Drug Administration (FDA) approved a gene replacement therapy for the inherited, progressive neuromuscular disease 5q-linked spinal muscular atrophy (SMA). Approval included all children with SMA under the age of two years; however, the gene therapy had only been studied in children aged up to 8 months. Now, a new study discusses safety and early outcomes in a large cohort of SMA patients under the age of two years who were treated with gene therapy.

Read more at Pediatrics Nationwide: A Multicenter Look at Gene Therapy for Spinal Muscular Atrophy.