The FDA accepted uniQure's biologics license application for ifezuntirgene inilparvovec and granted it Priority Review, setting a target action date of May 1, 2027.
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.
Jeffrey Cohen, MD, professor of neurology and staff physician at the Cleveland Clinic Mellen Center for Multiple Sclerosis, discusses interim BREAKFREE-2 findings for zola-cel, a CD19-directed CAR T cell therapy, in relapsing and progressive MS.
Interim phase 1b/2a data showed all patients treated with high- or low-dose BB-301 for oculopharyngeal muscular dystrophy met formal response criteria.
Although ocular signs in LEMS are typically mild and appear late, recognizing them alongside proximal weakness and autonomic symptoms can speed diagnosis and earlier detection of an associated lung cancer.