Marisa Wexler, MS, senior science writer —

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…

Note: This story was updated March 10, 2023, to correct a secondary headline to note the therapy was tracked for less than a year. The story also clarifies the timing of results from a Phase 3 clinical trial and that edaravone is believed to reduce oxidative stress. Radicava ORS, an…

The gene expression profiles of motor neurons — the specialized nerve cells that control movement and are lost in amyotrophic lateral sclerosis (ALS) — vary among men and women, with substantial differences found between the sexes in a new study done using induced pluripotent stem cells, or iPSCs. “The…

A team of U.S. researchers have identified two proteins in the lab — PIKFYVE and SYF2 — that may each be a useful therapeutic target for treating amyotrophic lateral sclerosis (ALS). Their findings on these potential molecular targets were published in a pair of studies earlier this month. “Our…

A group of experts, led by scientists from the National Institute of Neurological Disorders and Stroke (NINDS), has published a set of strategic priorities for the amyotrophic lateral sclerosis (ALS) community that are expected to guide future research funding. The goals include better understanding the biology underlying ALS, finding…