Marisa Wexler, MS, senior science writer —

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…

People with amyotrophic lateral sclerosis (ALS) who have a more active innate immune system — the part of the immune system that acts as a first responder to threats — tend to have faster disease progression and poorer survival, a study of large data showed. Findings suggest that measuring…

People with amyotrophic lateral sclerosis (ALS) spend time less asleep and wake up often during the night, a new analysis shows. Because good sleep is critical for quality of life, the findings emphasize the importance of testing sleep quality in ALS so appropriate treatments can be given if needed,…

New Brunswick is reimbursing, through its public health insurance program, the cost of Albrioza (sodium phenylbutyrate and ursodoxicoltaurine) for eligible residents living with amyotrophic lateral sclerosis (ALS). The Canadian province is the country’s fourth to provide public coverage for the oral therapy — marketed in the U.S.

It’s long been known that some molecules are misplaced in the nerve cells that progressively die in amyotrophic lateral sclerosis (ALS), but a new study shows this affects hundreds of different molecules, not just a handful, as previously thought. “This research represents a shift in our thinking about what…