News

Verge Genomics has launched a proof-of-concept trial to test whether VRG50635, its oral PIKfyve inhibitor therapy, can be used as a potential treatment for people with amyotrophic lateral sclerosis (ALS). The Phase 1b study will assess the safety and tolerability of escalating doses of VRG50635 — which works…

Researchers have unveiled a new AI-powered avatar platform — one making use of artificial intelligence (AI) technology — that aims to help people with amyotrophic lateral sclerosis (ALS) to communicate, even when the neurodegenerative disease progresses to the point that speaking and moving become difficult or impossible. The platform…

After two meetings with the U.S. Food and Drug Administration (FDA), Coya Therapeutics says it’s received constructive feedback — and has reached an alignment with the agency regarding development plans for COYA 302, its therapy candidate for people with amyotrophic lateral sclerosis (ALS). The company held the two…

Arbor Biotechnologies and 4DMT — two U.S. companies working in next-generation genetic medicines — have established a strategic partnership to develop and commercialize novel gene therapies for neurological diseases, including amyotrophic lateral sclerosis (ALS). The agreement includes the codevelopment of up to six product candidates for diseases…

PharmAust announced that it soon will engage with U.S. Food and Drug Administration (FDA) regulators regarding development plans for monepantel, its investigational therapy for amyotrophic lateral sclerosis (ALS) and motor neuron disease (MND). If the pre-investigational new drug meeting goes well, the company intends to file a…

BrainStorm Cell Therapeutics has received patents in Europe, Australia, and Israel covering the use of investigational NurOwn and NurOwn exosomes in treating amyotrophic lateral sclerosis (ALS) and other neurological disorders. The European patent specifically covers the use of an isolated population of mesenchymal stem cells —…

A previously unknown mutation in the SPTLC2 gene was identified in two patients who developed juvenile-onset amyotrophic lateral sclerosis (ALS), a study reports. The mutation significantly increased the production of certain types of fat-like molecules called sphingolipids, resulting in early-onset muscle weakness, progressive motor impairment, and involuntary tongue movements.

Researchers have developed an algorithm to generate personalized survival predictions for people with amyotrophic lateral sclerosis (ALS) based on clinical and MRI features. The scientists believe the algorithm could help patients and doctors make better treatment and care plans. “The disease progression varies greatly for ALS patients, so it…

Axoltis Pharma‘s new treatment candidate for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases was deemed safe and well tolerated at multiple doses in healthy volunteers who participated in a Phase 1b trial. Findings from the trial and preclinical studies also indicate NX210c is working as intended, boosting…

Throughout 2023, ALS News Today brought you daily coverage of the latest clinical research and scientific breakthroughs related to amyotrophic lateral sclerosis (ALS). Here are the year’s top 10 most-read articles, each with a brief description. We’re excited to remain a dependable resource for the ALS community in…