Steve Bryson, PhD,  science writer—

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…

QurAlis has secured $88 million to advance the clinical development of QRL-101 and QRL-201, its lead candidates for amyotrophic lateral sclerosis (ALS). The new series B financing, a second round of funding after the company met certain research milestones, brings the total investment to $143.5 million. The proceeds will…

Significant delays in the diagnosis of amyotrophic lateral sclerosis (ALS) were revealed by a new large-scale analysis of real-world patient records using artificial intelligence (AI). In fact, the median time from the first symptom to an ALS diagnosis was 11 months for these patients. Similar delays were seen regardless…

Researchers have characterized three molecular subtypes of amyotrophic lateral sclerosis (ALS), each one associated with different clinical outcomes, including age of disease onset, disease duration, and survival. These subtypes are based on unique patterns of gene activity in patient brain tissue and were independent of where the onset of…

A new pig model of amyotrophic lateral sclerosis (ALS) more accurately reflects the gradual motor neuron loss and disease progression seen in patients, and it may speed the development of ALS treatments, included gene and cell-based therapies, researchers report. The model carries a mutation in the SOD1 gene, an…