Treatment with the investigational therapy DNL343 was generally well-tolerated among people with amyotrophic lateral sclerosis (ALS) in an early clinical trial, and biomarker data from the trial suggest that the therapy is working as intended. Results were presented at the annual meeting of the American Academy of Neurology (AAN),…

An immune protein that is mostly produced in neurons was found to drive damage to mitochondria, which produce energy for cells, and nerve fiber loss, a study has found. Suppressing the protein, called gasdermin-E (GSDME), prevented nerve fiber loss in cells derived from people with amyotrophic lateral sclerosis (ALS),…

A first patient has been dosed in a Phase 1 clinical trial evaluating QRL-201, an experimental antisense oligonucleotide (ASO) molecule in treating amyotrophic lateral sclerosis (ALS), the therapy’s developer, QurAlis, announced. Dubbed ANQUR (NCT05633459), the study is reported to be the first into a potential therapy…

AI Therapeutics‘ experimental therapy AIT-101 led to reductions in levels of toxic proteins in people with amyotrophic lateral sclerosis (ALS) associated with mutations in the C9ORF72 gene, according to data from a clinical trial. The Phase 2a clinical trial also met its main goals of showing that AIT-101…

TDP-43, a protein that is known to have toxic effects in amyotrophic lateral sclerosis (ALS), is able to interact with RNA molecules carrying a chemical modification called m6A, and this chemical modification is increased in ALS nerve cells, a new study shows. The findings add to a growing body…

A Phase 3b study evaluating the long-term safety and effectiveness of two dosing regimens of Radicava ORS (edaravone) — an approved oral formulation of the medicine — in people with amyotrophic lateral sclerosis (ALS) is now fully enrolled. The study was designed to fulfill a post-marketing commitment that…

The U.S. Food and Drug Administration (FDA) will hold an advisory committee meeting to discuss BrainStorm Cell Therapeutics‘ application to approve NurOwn for amyotrophic lateral sclerosis (ALS). Such a meeting, called an ADCOM, is normally for when a therapy is under regulatory review so experts can discuss its…

On average, a person with amyotrophic lateral sclerosis (ALS) will require a wheelchair to get around within two years of the initial onset of disease symptoms. Findings underscore the condition’s rapid advance, and the need for new ALS treatments that work to slow disease progression, its researchers noted.

On a bitterly cold morning in New York last weekend, I woke before the sun and walked through the empty city streets to the Times Square subway station. From there, I caught a train to Brooklyn, the starting line for the United Airlines NYC Half, where more than…

Changes in neurofilament light chain (NfL) levels — a biomarker of nerve cell damage — were deemed by an advisory committee reasonably likely to predict clinical efficacy from tofersen in people with amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations. The unanimous vote from the Peripheral and Central Nervous…