Marisa Wexler, MS, senior science writer —

Note: This story was updated March 6, 2024, to correct the poster was presented by Brainstorm’s executive vice president and chief development officer Bob Dagher. A pivotal, Phase 3b clinical trial is planned to test the cell-based therapy NurOwn (debamestrocel) in people with amyotrophic lateral sclerosis (ALS),…

While the most recent guidelines for treating amyotrophic lateral sclerosis (ALS) recommend that patients receive three available treatments together, as soon as possible, a new analysis of U.S. data shows that most individuals with the progressive disease don’t get this combination in clinical practice. The analysis found that most…

Health Canada has issued a notice of noncompliance-withdrawal saying it won’t approve the oral therapy masitinib as an add-on treatment for amyotrophic lateral sclerosis (ALS). Its developer, AB Science, can submit a request for reconsideration within the next month, which it plans to do, it announced in…

An experimental gene therapy called CTx1000, which is designed to reduce toxic buildups of the protein TDP-43 in nerve cells, was found to significantly extend survival and improve motor function in mouse models of amyotrophic lateral sclerosis (ALS) and the related disorder frontotemporal dementia, known as FTD. According to…

Treatment with the experimental oral therapy ATH-1105 significantly reduced nerve damage, improved motor function, and prolonged survival in preclinical models of amyotrophic lateral sclerosis (ALS), a study reports. The work was funded by ATH-1105’s developer Athira Pharma, which plans to move the…

A new compound that significantly extended survival in a mouse model of amyotrophic lateral sclerosis (ALS) by blocking the activity of a group of proteins called MAP4Ks has been identified by researchers. The molecule was associated with modest benefits, but researchers believe it can…

Reduced activity of the TDP-43 protein, a hallmark of amyotrophic lateral sclerosis (ALS), leads to changes in the DNA of nerve cells, which alters the activity of important genes, a new study reports. These findings may help explain how problems with this protein can contribute to the death of…

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…

Scientists at King’s College London have identified the site in motor neurons that appears to be the starting point of dysfunction in amyotrophic lateral sclerosis (ALS) — shedding new light on the workings of the neurodegenerative disease at its earliest stages. “This provides a better understanding of the complex…

Amylyx Pharmaceuticals is developing AMX0114, a new experimental treatment for amyotrophic lateral sclerosis (ALS) that’s designed to promote the health of nerve fibers by lowering levels of calpain-2, a protein. AMX0114 is being tested in preclinical studies with the aim of supporting an investigational new drug application (IND),…