10 Years of Progress: A Timeline of FDA Approvals for Duchenne Muscular Dystrophy

Prior to 2016, Duchenne muscular dystrophy (DMD) had no disease-targeted treatment options and at the time, clinicians could offer corticosteroids to slow functional decline but nothing that addressed the underlying dystrophin deficiency driving the disease. That changed in the years since; the FDA has approved a steadily growing roster of exon-skipping therapies, a novel corticosteroid, a first-in-class HDAC inhibitor, and the field's first gene therapy.