Steve Bryson, PhD, science writer —

The Committee for Medicinal Products for Human Use (CHMP) has issued an opinion opposing Amylyx Pharmaceuticals’ bid for conditional European Union approval of AMX0035, its oral treatment to slow the progression of amyotrophic lateral sclerosis (ALS). The negative recommendation from the advisory committee, an arm of the…

Magnetic stimulation restored the health of lab-grown motor neurons — the muscle-controlling nerve cells that die in amyotrophic lateral sclerosis (ALS) — derived from people with familial ALS, according to a proof-of-concept study. The approach improved several biological processes in motor neurons, including the movement of cellular components along…

PEG10, an ancient, virus-like protein that supports the development of the placenta, appears to also participate in the nerve cell loss associated with amyotrophic lateral sclerosis (ALS), researchers discovered. ALS-causing mutations in the UBQLN2 gene were found to prevent the proper degradation of PEG10. This led to a…

MRI scans of the brains of people with amyotrophic lateral sclerosis (ALS) have identified a neurochemical called N-acetyl aspartate (NAA) whose levels correlate with the loss of motor function, a study reports. A drop in NAA levels was associated with the breakdown in communication between the primary motor cortex…

Health Canada has resumed its review of AB Science’s application seeking approval of masitinib, an oral add-on therapy for amyotrophic lateral sclerosis (ALS), after the agency halted its review late last year. In 2022, the agency agreed to review the application under the notice of compliance with…

Masitinib as an oral add-on therapy to Rilutek (riluzole) showed the greatest benefits in amyotrophic lateral sclerosis (ALS) patients with mild or moderate disease severity, an analysis of trial data showed. The findings come from the now-complete AB10015 Phase 2/3 clinical trial (NCT02588677) that excluded people…

An immune protein that is mostly produced in neurons was found to drive damage to mitochondria, which produce energy for cells, and nerve fiber loss, a study has found. Suppressing the protein, called gasdermin-E (GSDME), prevented nerve fiber loss in cells derived from people with amyotrophic lateral sclerosis (ALS),…

AB Science has submitted its response to the European Medicines Agency (EMA), part of an ongoing review process aiming for the conditional approval of masitinib as an add-on oral therapy for amyotrophic lateral sclerosis (ALS). The Committee for Medicinal Products for Human Use (CHMP), an arm…

Cytokinetics announced that it is stopping COURAGE-ALS, a Phase 3 trial investigating oral reldesemtiv in treating people with amyotrophic lateral sclerosis (ALS), due to a lack of efficacy compared with a placebo. In a review of unblinded interim trial findings at a second planned data monitoring committee meeting,…

Anew Medical‘s targeted gene therapy candidate, ANEW-202, eased multiple mechanisms associated with amyotrophic lateral sclerosis (ALS), leading to improved muscle function and survival in a mouse model of the disease. The gene therapy, licensed on an exclusive worldwide basis from the Autonomous University of Barcelona, in Spain, is…