Phase 4 Study Shows Stable Motor Function in Adults With SMA on Risdiplam Isabella Ciccone, MPH

Published Date: 03 Oct 2026

Real-world 12‑month results from WeSMA, a phase 4 study, showed that oral risdiplam is well tolerated in adults with SMA, with stable motor function and consistent safety findings.

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CSF Biomarkers Identify Shared LRRK2 Pathway Activation in Sporadic and LRRK2-Associated Parkinson Disease Louie Pasculli

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Todays Updates

Solengepras Meets Primary Endpoint in Phase 3 ARISE Trial of Parkinson Disease Marco Meglio

Phase 3 ARISE trial results show solengepras 150 mg significantly reduced OFF time and improved motor and non-motor symptoms in Parkinson disease.

Upadacitinib Feasible for Palmoplantar Pustulosis: David Gleeson, BM BCH David Gleeson, BM BCH

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Latest News

New cerebrospinal fluid biomarker data suggest shared LRRK2 pathway activation across sporadic and LRRK2-associated Parkinson disease, potentially informing biomarker development and therapeutic targeting.

Phase 2a trial results showed 800 mg vutiglabridin significantly improved motor symptom scores compared with placebo in early-stage Parkinson's disease.

An MDS 2026 plenary speaker discusses the evolving role of infusion therapies, deep brain stimulation, and focused ultrasound in Parkinson disease, including when to consider device-aided therapies and how treatment selection can be individualized.

Mitzi Joi Williams, MD, is joined by Jakai McEwen, DO, and Charnetta Colton-Poole, MD, to discuss the challenges of communication between patients and their health care teams, including navigating medical language, promoting shared decision-making, and advocating for clear, patient-centered care.

Christen Kutz, PhD, PA-C, discusses a retrospective survey of neurology providers examining why patients with generalized myasthenia gravis are transitioning to nipocalimab and what real-world outcomes data suggest about its role in the treatment landscape.

Carlos Cruchaga, PhD, director of the NeuroGenomics and Informatics Center at Washington University, discusses emerging blood-based biomarkers for monitoring biological and cognitive responses to lecanemab treatment in Alzheimer disease.

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LRRK2 Inhibitor BIIB122 Fails to Slow Early-Stage Parkinson Disease Marco Meglio

Full results from the phase 2b LUMA showed that investigational BIIB122/DNL151 did not slow confirmed clinical worsening in early-stage Parkinson's disease compared with placebo.

Phase 1 Data Link ARV-102 LRRK2 Degradation to Oculomotor and CSF Biomarker Changes in Parkinson Disease Louie Pasculli

Early phase 1 findings showed that the PROTAC LRRK2 degrader ARV-102 was associated with improvements in saccadic hypometria and changes in CSF markers of endolysosomal function, neuroinflammation, synaptic integrity, and axonal guidance in participants with Parkinson disease.

Nipocalimab’s Safety Profile Slightly Favored Over Other Myasthenia Gravis Therapies, Indirect Comparison Shows Marco Meglio

A new Bayesian network meta-analysis found nipocalimab's serious adverse event rates compared favorably with other gMG therapies, while overall adverse event rates were comparable.

Givinostat Data Point to Delayed Loss of Ambulation, Preserved Muscle Tissue in DMD Isabella Ciccone, MPH

An updated open-label extension analysis and new MRI data from the phase 3 EPIDYS trial, presented at the 2026 AANEM Annual Meeting, add to the evidence for givinostat's long-term effects on disease progression.

NeurologyLive® Brain Games: October 4, 2026 Louie Pasculli

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 the history of MDS!

Early Spinal Muscular Atrophy Treatment Tied to Milder Pediatric Phenotype Marco Meglio

New data from a Cure SMA survey showed that children treated with disease-modifying therapy soon after birth walk more often and have fewer comorbidities than those treated later.

Trappsol Cyclo Misses Primary End Point in Phase 3 Trial for Niemann-Pick Disease Type C Isabella Ciccone, MPH

Findings from the phase 3 TransportNPC study showed that Trappsol Cyclo missed the primary end point in Niemann-Pick type C, but subgroup and survival data support planned 2026 NDA.

NeurologyLive® Friday 5 — October 2, 2026 Isabella Ciccone, MPH

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 2, 2026.

Congress Passes ACT for ALS Reauthorization, Extending Research Programs Louie Pasculli

The Accelerating Access to Critical Therapies for ALS Reauthorization Act of 2026 extends federal support for ALS research and expanded access to investigational therapies, with additional provisions addressing clinical trial oversight and regulatory planning.

2026 Rachel Horne Prize Winner Sandra Vukusic, MD, PhD, on Advancing Women's Health in MS Isabella Ciccone, MPH, Sandra Vukusic, MD, PhD

The professor of neurology at the University of Lyon and 2026 Rachel Horne Prize Winner discussed pregnancy counseling, therapeutic inertia, and overlooked women's health issues in multiple sclerosis.

Phase 4 Study Evaluates Transition From IVIG to Subcutaneous Efgartigimod in CIDP Louie Pasculli

A phase 4 study is evaluating whether adults with chronic inflammatory demyelinating polyneuropathy can transition from intravenous immunoglobulin to subcutaneous efgartigimod PH20 without first experiencing disease worsening.

Phase 4 OCTAGON Trial to Test Rapid Steroid Taper With Ravulizumab Marco Meglio

A new phase 4 trial, OCTAGON, will test a rapid, predefined corticosteroid-tapering schedule in patients with generalized myasthenia gravis treated with ravulizumab.

LGMD Agent BBP-418 Improves Ambulation and Pulmonary Function Across Subgroups in Phase 3 FORTIFY Interim Analysis Isabella Ciccone, MPH

New 12-month interim data showed functional gains with BBP-418 vs placebo, consistent benefit across genotype, age, and baseline pulmonary function subgroups, and a safety profile comparable to placebo.

FDA Accepts Fenebrutinib NDA Under Priority Review for Relapsing and Primary Progressive MS Isabella Ciccone, MPH

The FDA accepted fenebrutinib's new drug application under priority review for relapsing and primary progressive MS, based on 3 phase 3 trials comparing the BTK inhibitor with teriflunomide and ocrelizumab.

ADHERE REAL Study to Assess Real-World Effectiveness of Efgartigimod in CIDP Isabella Ciccone, MPH

A new study will follow adults with chronic inflammatory demyelinating polyradiculoneuropathy who start subcutaneous efgartigimod in routine practice in the US and Germany.

Episode 176: BBP-418 and the Changing Treatment Landscape of LGMD Marco Meglio, Katherine Mathews, MD

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Katherine Matthews, MD. [LISTEN TIME: 17 minutes]

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