Feature|Articles|August 27, 2026

Evaluating Once-Yearly Salanersen Across the Spinal Muscular Atrophy Spectrum: A Phase 3 Clinical Program

Author(s)Mariane Hernandez, Isabella Ciccone, MPH

The phase 3 clinical program for salanersen, an investigational intrathecally administered antisense oligonucleotide, will test the efficacy of the agent across infants, adolescents and adults living with SMA.

Welcome to NeurologyLive's Clinical Trial in Focus. Every month, an ongoing clinical trial in the landscape of neurology is featured, highlighting the design of the study, the targeted patient population, the enrollment criteria, the primary and secondary end points, and its potential implications for clinical care.

Related to this article

October 14th, 2026

Arimoclomol Plus Miglustat Shows Early, Sustained Slowing of Niemann-Pick Disease Type C Progression

A post hoc analysis of the phase 2/3 NPC002 trial presented at CNS 2026 showed that the benefit of arimoclomol plus miglustat in Niemann-Pick disease type C emerged as early as 3 months after treatment initiation.

October 16th, 2026

RGX-202 Meets Primary End Point in Phase 3 Duchenne Trial

Pivotal AFFINITY DUCHENNE results showed microdystrophin expression above the prespecified threshold in 93% of evaluable participants, with preliminary functional findings favoring treatment over external controls.

October 17th, 2026

Nipocalimab in Myasthenia Gravis: Real-World Use, Long-Term Safety, and the Road Ahead

Leonard Dragone, MD, PhD, Disease Area Leader at Johnson & Johnson, discusses how nipocalimab is being incorporated into generalized MG care, what long-term data show, and where J&J sees the therapy’s role evolving.

October 16th, 2026

NeurologyLive® Friday 5 — October 16, 2026

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 16, 2026.

October 16th, 2026

Levacetylleucine Shows Sustained Benefit at 3 Years in Niemann-Pick Disease

Three-year extension data from the phase 3 IB1001-301 trial show levacetylleucine slowed disease progression in both adults and children with Niemann-Pick disease type C.

October 16th, 2026

FDA Approves Autoinjector for Self-Administered Zilucoplan in Generalized Myasthenia Gravis

Adults with generalized myasthenia gravis who are anti-acetylcholine receptor antibody positive now have a second self-administration option for the complement inhibitor zilucoplan.

October 16th, 2026

Episode 177: Previewing the 2026 NEALS Annual Meeting

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Nicholas J. Maragakis, MD; and Jinsy Andrews, MD, MSc, FAAN, FANA. [LISTEN TIME: 23 minutes]

October 15th, 2026

New Phase 1 Study Tests Intrathecal Gene Therapy Agent INS1201 in Duchenne Muscular Dystrophy

The phase 1 ASCEND trial is testing a single intrathecal dose of the investigational AAV9 gene therapy INS1201 in young boys with Duchenne muscular dystrophy.