Marisa Wexler, MS, senior science writer —

In a nearly unanimous vote, an advisory committee to the U.S. Food and Drug Administration (FDA) said there’s not enough evidence to support the efficacy of the experimental stem cell therapy NurOwn as a treatment for amyotrophic lateral sclerosis (ALS). The committee voted on a single question in its meeting: “Do…

The U.S. Food and Drug Administration (FDA) has shared its major concerns with data available for NurOwn, BrainStorm Cell Therapeutics‘ experimental stem cell therapy for amyotrophic lateral sclerosis (ALS). According to a new briefing document put out by the FDA, the agency believes that currently…

Every person with amyotrophic lateral sclerosis (ALS) should be offered genetic testing to check for the most common ALS-causing mutations, according to a new set of expert guidelines. “These evidence-based, consensus guidelines will support all stakeholders in the ALS community in navigating benefits and challenges of genetic testing,”…

PathMaker Neurosystems has launched a Phase 1 clinical trial that will test its experimental noninvasive nerve-modulating device in people with amyotrophic lateral sclerosis (ALS). This is the first time that the device — called the MyoRegulator and designed to slow disease progression in ALS — will…

Treatment with Ultomiris (ravulizumab-cwvz) — a therapy approved for several rare diseases that works by blocking activation of part of the immune system called the complement cascade — failed to slow the progression of amyotrophic lateral sclerosis (ALS) in a Phase 3 clinical trial that had been launched…

Bryostatin-1, a molecule being developed to treat amyotrophic lateral sclerosis (ALS), improved cell survival in a lab model of the disease, a new study reports. The therapy is known to activate PKC-epsilon, a protein that’s significantly reduced in ALS patients’ motor cortex, a brain region involved in muscle movement…

When the TDP-43 protein is less able to form two-protein complexes called dimers, it appears to be more likely to form the toxic clumps seen in amyotrophic lateral sclerosis (ALS), a study reports. As these clumps are found in about 97% of all ALS cases, a better understanding of the…

Mutations in the C9ORF7 gene, the most common genetic cause of amyotrophic lateral sclerosis (ALS), lead to defects in the stem cells needed for brain development in the early stages of life, a new study shows. In animal models of the disease, this resulted in smaller than normal volumes…

Scientists have devised a technique for transplanting healthy motor neurons — the nerve cells that are lost in amyotrophic lateral sclerosis (ALS) — in a mouse model with “highly aggressive” disease. Transplanted motor neurons, given stimulation, were able to form healthy connections with muscle cells to control the animals’ muscle…

Mutations in the gene NEK1, a major genetic cause of amyotrophic lateral sclerosis (ALS), may work to drive the disease by destabilizing the structure of nerve fibers and disrupting the movement of molecules inside nerve cells, according to a new study. The findings, which show for the first time how…