Marisa Wexler, MS, senior science writer —

The U.S. Food and Drug Administration (FDA) is funding several novel research projects related to amyotrophic lateral sclerosis (ALS) and other rare neurological disorders through its orphan drug grants program. In total, the regulatory agency has awarded 19 new grants and two contracts, providing more than $38 million…

A cholesterol-related protein, called apolipoprotein A1 (ApoA1), can prevent the death of endothelial cells — those lining blood vessel walls — in a cell model of amyotrophic lateral sclerosis (ALS), a recent lab study shows. According to researchers, these results “show promise for ApoA1 as a therapeutic agent to…

The nonprofit EverythingALS is teaming up with several biotech companies for a study into the feasibility of monitoring amyotrophic lateral sclerosis (ALS) progression in people via disease-specific biomarkers captured digitally at the home. The study will be open to adults with an ALS diagnosis or for whom ALS…

Helixmith has unveiled its new multi-approach program called DART — Defeating ALS through Regenerative Therapeutics — to develop new therapies for amyotrophic lateral sclerosis (ALS). Company CEO Sunyoung Kim presented the program in person and virtually at the 2022 Cell & Gene Meeting on the Mesa, Oct. 11–13.

There is no compelling evidence to suggest any medication increases the risk of developing amyotrophic lateral sclerosis (ALS), according to a review of published studies. If anything, classes of drugs including anti-hypertensives, cholesterol-lowering statins, aspirin, and oral contraceptives may be associated with a lower risk of ALS, although several…

Early treatment with Biogen’s experimental therapy tofersen slows disease progression in people with amyotrophic lateral sclerosis (ALS) associated with SOD1 gene mutations, compared with patients who started treatment after a six-month delay, according to findings from a Phase 3 trial and its open-label extension study. These benefits…

PrimeC, an experimental combination treatment for amyotrophic lateral sclerosis (ALS) being developed by NeuroSense Therapeutics, was well tolerated and lowered disease biomarkers in an open-label Phase 2 clinical trial. That’s according to the study, “Combination of ciprofloxacin/celecoxib as a novel therapeutic strategy for ALS,” published…

AMX0035 — Amylyx Pharmaceuticals’ experimental combination therapy for amyotrophic lateral sclerosis (ALS) now under review for approval in the U.S. — alters the activity of genes involved in several disease-relevant pathways, according to a study in lab-grown cells. Notably, the two-compound oral treatment was seen to change the activity of many…

The levels of five small RNA molecules, called microRNAs, inside tiny vesicles in the blood of people with amyotrophic lateral sclerosis (ALS) differ significantly from those of people without ALS and may offer a way of accurately and quickly diagnosing the disease, a study suggests. Importantly, its researchers evaluated…

A treatment known as VPA-Li, a combination of lithium carbonate and valproic acid, may slow disease progression and improve quality of life for people with amyotrophic lateral sclerosis (ALS), according to data from a small Phase 2 clinical trial. Favorable trends in survival and lung health were also observed…