Stem cell therapy, specifically treatment with mesenchymal stem cells or MSCs, did not substantially alter disease progression among people with amyotrophic lateral sclerosis (ALS), according to new data from a Phase 2 clinical trial. However, there was a subset of patients who did appear to experience notable benefits from…
News
While the now-discontinued amyotrophic lateral sclerosis (ALS) therapy Relyvrio (sodium phenylbutyrate and taurursodiol) failed to show a meaningful effect on disease progression, lung function, or life quality in the Phase 3 PHOENIX clinical trial, ongoing analyses will look at other outcomes such as biomarkers and survival. That’s according…
Storing chemicals such as gasoline, lawn care products, and paints in a garage attached to a home was associated with an increased risk of amyotrophic lateral sclerosis (ALS) in a recent analysis. Researchers believe the findings add to knowledge about environmental exposures — workplace, recreational, or residential — that…
Columbia University researchers will use a $15 million federal grant to design individualized gene therapies for nine people with ultra-rare genetic forms of amyotrophic lateral sclerosis (ALS). The three-year grant comes from the National Institute of Neurological Disorders and Stroke’s (NINDS) Ultra-rare Gene-based Therapy (URGenT) Network, a program…
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) showed that patients who received Relyvrio…
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