Two-year data from the phase 2 DAZALS trial showed a lower risk of death with dazucorilant 300 mg/day vs placebo in ALS, although the study did not meet its functional primary end point.
QurAlis reported interim ANQUR trial results showing target engagement, a biomarker signal, and a trend toward slower functional decline with QRL-201 in ALS.
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.