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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.

The FDA clearance allows AbelZeta to study bispecific CAR T-cell therapy C-CAR168 in refractory progressive multiple sclerosis.

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.

Alit Stark-Inbar, PhD, Chief Scientific Officer of Theranica, writes about migraine prevention beyond lifestyle changes, highlighting the role of neuromodulation and other evidence-based treatments in long-term management.

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.

Michela Leocadi, PhD, research fellow at UCL Queen Square Institute of Neurology, discussed new longitudinal data showing early functional connectivity changes in HD gene carriers years before clinical motor diagnosis.

In a phase 4 randomized controlled trial, opicapone failed to demonstrate statistical superiority over placebo for reducing fluctuation-related pain in patients living with Parkinson disease.

KuangHua Guo, MD, PhD, sleep specialist at Northwestern Medicine, discussed emerging orexin agonist therapies and refined diagnostic approaches for hypersomnia.

Microstructure Imaging has announced that the FDA cleared its MICSI-PET, an MR-guided PET enhancement platform with automated amyloid Centiloid and tau quantification.

Subgroup analyses from the phase 2 ADDRESS-LC trial showed bezisterim significantly improved fatigue, malaise, and cognition in long COVID patients with more severe baseline symptoms.

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