Lindsey Shapiro, PhD,  science writer—

The investigational therapy BIIB078 was generally well tolerated in a Phase 1 clinical trial, but did not show evidence of slowing disease progression in people with amyotrophic lateral sclerosis (ALS) caused by mutations in the C9orf72 gene, according to recently published data from the trial. Because the trial failed to…

Clene said it planned to give the U.S. Food and Drug Administration (FDA) new data on CNM-Au8 for the treatment of amyotrophic lateral sclerosis (ALS) ahead of a meeting on a path to accelerated approval of the therapy. The company said it would submit a briefing book…

Researchers have identified gene activity changes that might explain why motor nerve cells selectively degenerate in amyotrophic lateral sclerosis (ALS), offering insights into the development of new therapeutic targets for the neurodegenerative disease. In postmortem brain tissue, a particular group of these cells known to be lost in ALS…

New research into the molecular underpinnings of amyotrophic lateral sclerosis (ALS) has uncovered sex differences and disease subtypes, which could inform the development of new and more targeted treatment strategies. Comprehensive analyses of ALS brain tissue samples and mouse models also identified the MAPK/ERK cell signaling pathway as a possible…

Men who are more active and physically fit have lower long-term risk of developing amyotrophic lateral sclerosis (ALS), according to findings from a large Norwegian study. No such link was observed in female patients, for reasons that are not entirely clear. The results may counter a concern that higher…

Amyotrophic lateral sclerosis (ALS) patients who used NeuroSense Therapeutics‘ investigational therapy PrimeC for a year saw significantly slower disease progression and prolonged survival relative to patients who started the treatment six months later. That’s according to one-year findings from the PARADIGM Phase 2b clinical trial (NCT05357950),…

Bravyl, an investigational oral therapy from Woolsey Pharmaceuticals, was safe and lowered levels of the nerve damage biomarker neurofilament light chain (NfL) in people with amyotrophic lateral sclerosis (ALS), according to new data from a Phase 2a clinical trial. The medication also showed early signs of slowing disease…

Adding Radicava (edaravone) to riluzole prolongs survival of amyotrophic lateral sclerosis (ALS) patients by more than three months relative to riluzole alone, according to analyses of real-world data from the ALS/MND Natural History Study Consortium database. These and other Radicava-related findings were presented by the therapy’s developer…

Increasing levels of the MIF protein may be a promising therapeutic strategy for slowing disease progression in amyotrophic lateral sclerosis (ALS), a study found. This approach in a mouse model of ALS with mutations in the SOD1 gene preserved motor function and prolonged survival, while lowering neuroinflammation and restoring…

The Foundation for the National Institutes of Health (FNIH) is launching a public-private partnership to generate the largest openly available data platform in amyotrophic lateral sclerosis (ALS) research, with the goal of accelerating the diagnosis and treatment of the progressive neurodegenerative disease. This five-year initiative will be conducted through…