Joanna Wardlaw, MD, Professor of Applied Neuroimaging at the University of Edinburgh, discusses the evolving understanding of cerebral small vessel disease, emerging therapeutic approaches, and the makings behind a successful trial.
QurAlis reported interim ANQUR trial results showing target engagement, a biomarker signal, and a trend toward slower functional decline with QRL-201 in ALS.
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.