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Coave Therapeutics has received a grant from the ALS Association to support the development of its gene therapy candidate CTx-TFEB for amyotrophic lateral sclerosis (ALS). The grant, made through the Lawrence and Isabel Barnett Drug Development Program, is part of the $2.9 million the ALS Association…

Daily treatment with monepantel — a drug widely used in veterinary medicine — was shown to reduce the rate of disease progression by as much as 58% in people with amyotrophic lateral sclerosis (ALS) or motor neuron disease (MND), according to top-line data from a Phase 1 trial.

Blocking a protein called PTP sigma led to better nerve survival and motor function in a mouse model of amyotrophic lateral sclerosis (ALS) caused by mutations in the C9ORF72 gene — leading the researchers to highlight this strategy for potentially treating this genetic ALS form. Data from cell models…

Relyvrio (sodium phenylbutyrate and taurursodiol), an approved treatment for people with amyotrophic lateral sclerosis (ALS), has failed to meet its primary goal of slowing disease progression in an ongoing Phase 3 clinical trial designed to confirm its effectiveness. That’s according to new data from the trial, dubbed PHOENIX,…

New data show that blood-based measures, including levels of markers of nerve damage and chemical DNA modifications, can help predict which individuals with amyotrophic lateral sclerosis (ALS) will have faster disease progression over time. However, because the disease course may be altered with Radicava or Radicava ORS —…

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…

Treatment with NeuroSense Therapeutics’ PrimeC was found to extend patients’ time without complications or death, and to lead to clinically meaningful effects on quality of life, among those with amyotrophic lateral sclerosis (ALS). The new data comes from the PARADIGM Phase 2b study (NCT05357950), a fully enrolled…

OrphAI Therapeutics’ experimental therapy AIT-101 has been awarded orphan drug status in the European Union for amyotrophic lateral sclerosis (ALS), following a similar designation granted in the U.S. last year. In Europe, orphan drug status is given to medicines designed to treat life-threatening or chronically debilitating conditions affecting…

Treatment with CNM-Au8 was shown to significantly prolong survival — relative to the natural history of the disease — among people with advanced amyotrophic lateral sclerosis (ALS) who took part in two expanded access programs (EAP), also known as compassionate use programs. The findings from these EAPs, which…