Marisa Wexler, MS,  senior science writer—

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…

An expanded access program for the experimental treatment SLS-005 has dosed its first participant, according to Seelos Therapeutics, the therapy’s developer. The expanded access program, or EAP, is an open-label study allowing access to SLS-005 for people with amyotrophic lateral sclerosis (ALS) who are…

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…

Difficulties in measuring the progression of amyotrophic lateral sclerosis (ALS) among patients with advanced disease can cause complications in clinical trials, according to a new analysis that highlights how such problems may have affected the failed NurOwn study. The work was led by scientists at BrainStorm…

In most cases of amyotrophic lateral sclerosis (ALS), dysfunction of a protein known as TDP-43 leads to abnormally low levels of another protein, stathmin-2, which is sufficient to drive nerve cell dysfunction, new data suggest. In an aim to restore stathmin-2 levels, researchers created a designer DNA treatment. Now,…

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.

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…

A potential new genetic risk factor for amyotrophic lateral sclerosis (ALS) has been discovered by researchers at the University of Washington. The scientists say a mutation in which a region of the WDR7 gene is repeated more times than usual may increase the risk of ALS. “We believe that…

Four people with amyotrophic lateral sclerosis (ALS) each experienced a marked slowing in disease progression with COYA 302, an experimental immune-modulating therapy tested in a small proof-of-concept clinical trial, according to Coya Therapeutics, its developer. Based on these findings, Coya is planning to start work on further trials to…

Amyotrophic lateral sclerosis (ALS) patients treated with Radicava (edaravone) tend to go longer before reaching disease milestones such as needing a walking aid or breathing support, according to an analysis of insurance data. The findings were presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in…