Neuromuscular

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

In recognition of International Ataxia Awareness Day, observed annually on September 25, NeurologyLive® reviews 6 investigational agents in clinical development across ataxia-related disorders.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending September 25, 2026.

One-year KYSA-6 data showed KYV-101, a CD19 CAR T-cell therapy, produced durable, drug-free responses in generalized myasthenia gravis.

The FDA has granted Scholar Rock's apitegromab fast track designation for the treatment of patients with living facioscapulohumeral muscular dystrophy as the company's phase 2 FORGE study dosing begins.

Ulefnersen met its primary end point in the phase 3 FUSION trial, providing the first placebo-controlled evidence for a genetically targeted therapy in FUS-ALS.

The CHMP recommended approval of gefurulimab (Klygefa), a weekly, self-administered C5 inhibitor, for AChR-antibody-positive generalized myasthenia gravis in the EU.

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

The approval, based on phase 3 IB1001-303 trial data showing consistent SARA scale improvement, makes levacetylleucine the first treatment approved specifically for ataxia-telangiectasia.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending September 18, 2026.

Brian Lin, PhD, discussed the current evidence linking football participation with ALS, the questions that remain unanswered, and how emerging research may help clarify the association.

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.

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

Emerging therapies for muscular dystrophy are targeting a range of disease mechanisms, from RNA-based approaches and muscle-directed delivery to cell therapy and DUX4 suppression.

FDA approval of apitegromab introduces a muscle-targeting approach to SMA treatment, with phase 3 data demonstrating motor-function benefit when added to existing SMN2-directed therapies.

Rory Siegel, DO, discusses the emerging evidence linking football participation with ALS, the potential role of repetitive head impacts, and key questions that remain about long-term neurological risk.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending September 11, 2026.

Updated phase 1/2 POLARIS data showed sustained reductions in seizure frequency and progressive gains in cognitive and adaptive measures following a single administration of investigational ETX101 in children with SCN1A+ Dravet syndrome.