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…
AAN 2024: Trial investigator shares details from Relyvrio failure in ALS
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).
Last week, my kids and I drove from Michigan’s Upper Peninsula to the Milwaukee area for spring break. I have many fond memories of living there with my husband, Todd, more than a decade ago, before he got ALS. It’s too difficult for him to travel now due to…
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…
Adapt, adapt, adapt! Readers of this column know that’s one of my favorite mottos. I depend on it, especially because I live with ALS. It helps me deal with the condition’s ongoing changes and keeps me moving forward. But sometimes I have to push back. And when…
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…