Lindsey Shapiro, PhD, science writer —

Treatment with CNM-Au8 was found to significantly delay disease progression and prolong survival, by more than 1.5 years, among adults with early-stage amyotrophic lateral sclerosis (ALS) in the RESCUE-ALS trial and its open-label extension phase. That’s according to two different statistical analyses, which overall showed that patients who…

BL-001, an investigational gut-targeted therapy being developed for people with amyotrophic lateral sclerosis (ALS) and Dravet syndrome, was found to be safe and well tolerated among healthy volunteers in a Phase 1 clinical trial. Findings overall indicate that Bloom Science‘s BL-001 can be given safely to humans at…

Note: This story was updated Aug. 22, 2023, to correct there are four ALS approved therapies widely available in the U.S. Diarrhea associated with a common amyotrophic lateral sclerosis (ALS) treatment could be a substantial barrier to complying with the medication, according to surveys by Napo Pharmaceuticals. While…

Researchers have identified new genetic variants that might influence survival among sporadic amyotrophic lateral sclerosis (ALS) patients in Japan. Genetic analyses in more than 1,000 ALS patients uncovered that small changes in the FGF1, THSD7A, and LRP1 genes could affect patients’ prognosis. In patient-derived nerve cells, the identified variants led…

An earlier start to noninvasive ventilation (NIV) could help to improve respiratory function and prolong survival for people with amyotrophic lateral sclerosis (ALS), according to a small clinical trial in Spain. While many of the study’s findings failed to reach statistical significance, the researchers believe the data still support…

An into-the-nose (intranasal) formulation of edaravone — the active ingredient in the amyotrophic lateral sclerosis (ALS) medication Radicava — may enhance the medication’s ability to reach brain tissue, according to a recent study. The formulation, which was made by packaging edaravone into tiny carriers called nanoparticles, was found…

Albrioza, the approved treatment for amyotrophic lateral sclerosis (ALS) formerly known as AMX0035, is one giant step closer to being eligible for reimbursement under public drug plans in Canada, according to its developer, Amylyx Pharmaceuticals. Amylyx has reached an agreement with the pan-Canadian Pharmaceutical Alliance, or pcPA —…

Early treatment with ropinirole, a medication used to treat Parkinson’s disease, may slow disease progression in amyotrophic lateral sclerosis (ALS) patients, according to recently published data from a Phase 1/2 trial. Patients given the therapy showed slower functional declines and delayed disease progression over those who started treatment…

Mutations in the KIF5A gene, associated with some cases of amyotrophic lateral sclerosis (ALS), may lead to impairments in motor unit recovery that are exacerbated with age, according to a recent study. Motor units — nerve cells involved in movement and the muscle fibers they interact with — showed poor…

Locanabio‘s investigational therapy significantly reduced the buildup of toxic RNA molecules in amyotrophic lateral sclerosis (ALS) patient cells and mouse models associated with C9ORF72 mutations, according to new data. These RNA products — intermediate molecules that are produced from DNA as a template for protein production — are…