Marisa Wexler, MS, senior science writer —

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…

In a reversal, an advisory committee of the U.S. Food and Drug Administration (FDA) now says that the current evidence of efficacy of Amylyx Pharmaceuticals’ AMX0035 is sufficient to support its approval for amyotrophic lateral sclerosis (ALS). The committee voted 7–2 in favor of AMX0035, Amylyx announced…

Higher blood levels of antibodies against the viral protein HERV-K ENV are associated with a better survival rate and improved outcomes in people with amyotrophic lateral sclerosis (ALS), a new study shows. These findings further support the therapeutic potential of GNK301, an experimental antibody against HERV-K ENV being…

Altered forms of the SOD1 protein are found in the spinal cord nerve cells of people with all types of amyotrophic lateral sclerosis (ALS), not just those with mutations in the gene providing instructions for making the protein, a study reported. “The results suggest this abnormal protein contributes to cell…

Treatment with gene therapy candidate SynCav1 delayed disease onset and extended survival in a mouse model of amyotrophic lateral sclerosis (ALS), according to a new study. The experimental therapy aims to improve the survival of motor neurons in people with ALS irrespective of the underlying cause. “These data suggest that…

Treatment with terazosin, a medication approved to treat high blood pressure and enlarged prostate, was found to protect motor neurons and extend survival in animal models of amyotrophic lateral sclerosis (ALS) in a new study. “Our work shows that terazosin is protective of motor neuron cell death in multiple…