News

Treatment with Qalsody (tofersen) in the real world slowed disease progression in people with amyotrophic lateral sclerosis (ALS) caused by SOD1 mutations, a small study reports. It also stabilized patients’ quality of life and lowered levels of nerve damage-related biomarkers, which is consistent with clinical trial data that…

A double-blind Phase 2 clinical trial that will test Corcept Therapeutics’ oral cortisol modulator dazucorilant against a placebo in people with amyotrophic lateral sclerosis (ALS) is now fully enrolled, the company announced in a press release. Called DAZALS (NCT05407324), the trial involves 249 adults with sporadic…

Treatment with NurOwn (debamestrocel), which BrainStorm Cell Therapeutics is developing for amyotrophic lateral sclerosis (ALS), may bring about changes in biomarkers of inflammation and neurodegeneration that predict clinical outcomes. The findings come from the Phase 3 trial (NCT03280056) that tested NurOwn against a placebo in 189 adults with rapidly…

Troriluzole, a new formulation of the approved amyotrophic lateral sclerosis (ALS) treatment riluzole, can be taken with or without food and appears to have better pharmacological properties than approved versions of the medication that allow once daily administration. That’s according to data from three studies in healthy volunteers presented…

NeuroSense Therapeutics’ investigational therapy PrimeC tends to reduce blood levels of neurofilament light chain (NfL), a biomarker of nerve damage, in people with amyotrophic lateral sclerosis (ALS) — especially when given earlier in the course of the disease. That’s according to data from the PARADIGM Phase 2b…

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…

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).