Improvement in Management of Duchenne Muscular Dystrophy in Recent Years

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.

Marketed as Fayuvi, the 1-time AAV9 gene therapy showed sustained cerebrospinal fluid heparan sulfate drops and developmental gains among pediatric patients with mucopolysaccharidosis type IIIA in prior studies.

Amy Waldman, MD, Medical Director of the Leukodystrophy Center at Children's Hospital of Philadelphia, discusses zilganersen's mechanism, safety profile, clinical considerations, and what the first-ever approval for Alexander disease means for the broader leukodystrophy field.

Results recently published from the phase 2 HIMALAYA trial reported that SAR443820 did not slow functional decline, and was associated with more adverse events and treatment discontinuations in patients with ALS.

A new study reported that tremor and myoclonus affected nearly 7 in 10 patients with chronic inflammatory demyelinating polyneuropathy, with immunotherapy and beta-blockers showing potential benefit for symptom improvement.

Robert Bowser, PhD, chief scientific officer at Barrow Neurological Institute, recaps his ALS Nexus presentation on TDP-43 biology, the emerging therapeutic landscape, and how cryptic peptide biomarkers could reshape ALS clinical trial design.

Seven experimental DM1 therapies show promise and pitfalls; see latest trial data, a major phase 3 miss, and key 2026 readouts.

The FDA has approved zilganersen (Zanvastro), the first Alexander disease treatment, giving clinicians a disease-modifying option and a clearer framework for counseling patients across the full age and phenotype spectrum.