FDA Action Update, April 2026: Acceptance, Clearance, and Complete Response Letter

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.

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.

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.

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.