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CurePSP and UCSF announced enrollment of the first participant in the PSP Trial Platform, believed to be the first platform trial ever conducted specifically for progressive supranuclear palsy, designed to test multiple investigational drugs simultaneously.

In a recently published phase 3 post hoc analysis, treatment with fenfluramine was associated with early, sustained reductions in fall-related seizures among patients with Lennox-Gastaut syndrome.

Ractigen Therapeutics announced completion of enrollment and first dosing across all cohorts of its phase 2 trial of RAG-17, an investigational siRNA therapy for SOD1-mutated ALS, building on phase 1 data recently published in Nature Medicine.

Senda Ajroud-Driss, MD, director of the ALS Clinic at Northwestern Medicine, discussed why adults with spinal muscular atrophy continue to need multidisciplinary care and where the evidence still falls short.

Divya Jayaraman, MD, PhD, a pediatric neuromuscular specialist at Columbia University Irving Medical Center, discussed the comparative evidence gaps in spinal muscular atrophy and the difficulty of moving patients into adult care.

Horacio Kaufmann, MD, Director of the Dysautonomia Center at NYU Langone, discusses the prospective validation of prodromal MSA criteria, how olfactory testing can sharpen specificity, and how close the field is to biomarker-driven early diagnosis.

Annexon reported that all 10 patients in the first US and European cohort of its open-label FORWARD study showed rapid, clinically meaningful improvement in strength within days of a single tanruprubart infusion for Guillain-Barré syndrome.

Sandeep Rana, MD, director of the ALS Center at the Allegheny Health Network Neuroscience Institute, discussed how multidisciplinary clinics are assembled and what still limits access to coordinated care in spinal muscular atrophy.

Divya Jayaraman, MD, PhD, a pediatric neuromuscular specialist at Columbia University Irving Medical Center, discussed how multidisciplinary care teams in spinal muscular atrophy are adapting to a growing number of treatment options.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is Spinal Muscular Atrophy!

Investigators independently tracked the 6 patients treated in a Parkinson disease gene therapy trial that was abandoned mid-study after its sponsor became insolvent, finding a reassuring safety profile and mixed signals of motor benefit through up to 3 years.

Cynthia Keator, MD, director of neurology at Cook Children's Hospital, shares her perspective on building longitudinal Lennox-Gastaut syndrome care plans, navigating the pediatric-to-adult transition, and what the field needs to do better for patients and families.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 7, 2026.