Long-term Data with ITB Pumps for Spasticity

The 500-participant study will assess whether pridopidine, an investigational sigma-1 receptor agonist, can slow functional decline and improve survival in patients with early, rapidly progressing amyotrophic lateral sclerosis.

Kiril Kiprovski, MD, clinical professor of neurology and director of the Division of Neuromuscular Medicine at NYU Langone, reviews the clinical clues, bedside tests, and referral considerations that help eye care clinicians identify Lambert-Eaton myasthenic syndrome in patients with fluctuating ptosis or diplopia.

uniQure presented final safety and biomarker results from the discontinued EPISOD1 trial of AMT-162, an investigational gene therapy for SOD1-associated ALS.

Richard Nowak, MD, director of the Yale Myasthenia Gravis Clinic and global principal investigator of the MINT trial, discusses open-label extension findings for inebilizumab, a CD19-directed B cell depleting therapy, in generalized myasthenia gravis.

The executive medical director at AbbVie discussed the FDA approval of tavapadon, the first D1/D5 dopamine agonist for Parkinson disease, including the supporting TEMPO trial data and safety considerations.

Alnylam Pharmaceuticals reported initial Phase 1b findings showing brezosiran reduced cerebrospinal fluid mutant huntingtin levels by a mean of 60% to 64% at 3 months, with plans for a pivotal study in 2027.

AL-S Pharma outlined the design of AP101-03, a planned phase 3 trial of AP-101 in sporadic and SOD1-associated ALS, at the 2026 NEALS Annual Meeting.

Sabirnetug failed to meet its primary endpoint in Acumen Pharmaceuticals' phase 2 ALTITUDE-AD trial for early Alzheimer disease, prompting the company to end development.