Feature|Articles|September 17, 2026

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

Fact checked by: Marco Meglio

In honor of Muscular Dystrophy Awareness Month, held annually in September, NeurologyLive® looks back at the decade-long expansion of the DMD treatment landscape and the data that supports each approval.

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.

Related to this article

November 13th, 2026

Philadelphia Forum Explores Diagnostic Delays and Biologic Sequencing in Myasthenia Gravis

At a NeurologyLive Clinical Forum in Philadelphia, Chafic Karam, MD, chief of neuromuscular medicine at Penn Medicine, discussed treatment sequencing in myasthenia gravis.

November 12th, 2026

GV101 Gains FDA Fast Track Status for Friedreich Ataxia Ahead of Phase 2 Trial

GV101 receives FDA fast track designation for the treatment of Friedreich ataxia, with a phase 2 trial planned to begin enrollment in late 2026.

November 9th, 2026

FDA Investigates Confirmed Insertional Oncogenesis With Spinal Muscular Atrophy Gene Therapy

Postmarketing reports of product-related insertional oncogenesis involving GLI1 have prompted an FDA investigation into malignancy risk with SMA gene therapy, onasemnogene abeparvovec.

November 9th, 2026

T-Cell Engager Cizutamig Shows Early Efficacy Signal in Generalized Myasthenia Gravis

Interim phase 1b data showed that cizutamig produced rapid, sustained improvement in generalized myasthenia gravis with no cases of cytokine release syndrome.

November 9th, 2026

FDA Action Update, October 2026: Approval, Acceptance, and Designations

Catch up on any of the neurology headlines you may have missed in October 2026, compiled into 1 place by the NeurologyLive® team.

November 9th, 2026

This Week on NeurologyLive® — November 9, 2026

Here's some of what is coming soon to NeurologyLive® this week.

November 8th, 2026

PrimeC Lowers Neuron-Derived TDP-43, Informing Phase 3 PARAGON Trial in ALS

Newly presented biomarker and clinical findings from the phase 2b PARADIGM trial of PrimeC informed the design of the global phase 3 PARAGON trial in ALS.

November 7th, 2026

ALS Agent Dazucorilant Associated With Lower 2-Year Mortality in Exploratory DAZALS Analysis

Two-year data from the phase 2 DAZALS trial showed a lower risk of death with dazucorilant 300 mg/day vs placebo in ALS, although the study did not meet its functional primary end point.