The Muscular Dystrophy Association (MDA) is marking its seven-decade partnership with the International Association of Fire Fighters (IAFF) with this year’s launch of Fill the Boot, a nationwide yearlong fundraising campaign supporting research, care, and advocacy for people with neuromuscular disorders such as amyotrophic lateral sclerosis (ALS).
In people who experience a traumatic brain injury, or TBI — which is linked to a greater likelihood of developing amyotrophic lateral sclerosis (ALS) — blocking the activity of a protein called KCNJ2 may lower the risk of ALS, according to new research done in lab models. “Targeting KCNJ2…
The U.S. Food and Drug Administration (FDA) has agreed on the design of a Phase 3b trial that will test the investigational cell-based therapy NurOwn (debamestrocel) in amyotrophic lateral sclerosis (ALS) patients with less advanced disease. The developer, BrainStorm Cell Therapeutics, plans to launch the trial…
ASHA-624, an experimental therapy that blocks the activity of the SARM1 protein, will be developed as a potential disease-modifying therapy for amyotrophic lateral sclerosis (ALS). Developer Asha Therapeutics said it will conduct preclinical studies aimed at supporting the treatment’s potential for testing in human clinical trials. “As a veteran CNS…
The developer of the add-on treatment masitinib for amyotrophic lateral sclerosis (ALS) has been given the go-ahead to ask Health Canada to reconsider its decision from earlier this year against the approval of the oral therapy. The Canadian regulatory agency granted reconsideration eligibility to AB Science for…
Relyvrio (sodium phenylbutyrate and taurursodiol), an approved treatment for amyotrophic lateral sclerosis (ALS), was voluntarily removed from the U.S. and Canadian markets. Amylyx Pharmaceuticals, the therapy’s developer, made the decision after top-line results from the Phase 3 PHOENIX trial (NCT05021536)Â showed that patients who received Relyvrio…
SLS-005 did not significantly slow amyotrophic lateral sclerosis (ALS) progression compared with a placebo in the HEALEY platform trial, according to top-line data showing that the experimental therapy did not ease functional declines nor extend patient survival. The therapy, developed by Seelos Therapeutics, also failed to slow…
The U.S. Food and Drug Administration (FDA) has lifted a partial clinical hold that limited the use of certain dose levels of prosetin being tested in ProJenX’s Phase 1 trial. The PRO-101 trial (NCT05279755) is a three-part study designed to test the oral medication in healthy volunteers and…
Spanish pharmaceutical company Ferrer will co-develop and market Verge Genomics’ investigational PIKfyve inhibitor VRG50635 for the treatment of amyotrophic lateral sclerosis (ALS) in Europe, Central and South America, Southeast Asia, and Japan, under a collaboration agreement announced by the two companies. Ferrer will have exclusive co-development and commercialization…
Brain Trust Bio (BTB) has been cleared to launch Phase 1 clinical trials in Australia to test the efficacy of its patented method for delivering therapeutics directly into the spinal canal, with a focus on the amyotrophic lateral sclerosis (ALS) therapy riluzole. The approach is intended to further…
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