Partially lowering levels of the protein RAD23A can help nerve cells better handle toxic TDP-43 protein clumps, improving survival and function in a mouse model of amyotrophic lateral sclerosis (ALS), a study found. The approach reduced the buildup of misfolded proteins, restored the cell’s protein cleanup machinery, and slowed…
For many people with amyotrophic lateral sclerosis (ALS), even a slight slowing of disease progression is considered clinically meaningful, according to a new study based on a survey of hundreds of patients. “These results highlight the need for clinical trial designs that are capable of detecting small but meaningful…
Long-term exposure to air pollution, even at relatively low levels, is associated with an increased risk of developing motor neuron disease (MND), a group of conditions that includes amyotrophic lateral sclerosis (ALS), according to a new study by researchers in Sweden. Among patients, exposure to air pollution is also…
Target ALS has announced the launch of its redesigned website, which improves accessibility and highlights its research initiatives to accelerate the development of effective treatments for amyotrophic lateral sclerosis (ALS). The new digital platform offers an improved experience for the ALS community, facilitating access to more information about…
Scientists have developed a new computer-based model that mimics the spinal cord’s neural networks to predict how nerve cells degenerate in amyotrophic lateral sclerosis (ALS). Using this simulation, researchers discovered that targeting a specific group of “coordinator” cells, known as V1 interneurons, could prevent motor neuron death.
Dewpoint Therapeutics has selected a novel small molecule designed to prevent the formation of abnormal TDP-43 protein clumps that drive nerve cell death in amyotrophic lateral sclerosis (ALS) and other related conditions. The candidate is specifically designed to target condensates, or membrane-less structures that concentrate specific molecules to…
Overactivity of a molecular system that normally helps clear unwanted proteins may contribute to nerve cell damage in certain genetic forms of amyotrophic lateral sclerosis (ALS), a new study suggests. The findings may open new avenues for treatment, researchers wrote in the study titled “Aberrant nuclear…
Mindwalk Holdings has identified and validated antibodies that selectively target an abnormal form of the TDP-43 protein, which is found in about 97% of amyotrophic lateral sclerosis (ALS) cases and is also linked to other neurodegenerative diseases. The candidates include antibodies that could potentially be given as a…
The first patient has been dosed in a Phase 2 clinical study testing RAG-17, a therapy being developed by Ractigen Therapeutics for people with amyotrophic lateral sclerosis (ALS) who carry mutations in the SOD1 gene (SOD1-ALS). The progression into the Phase 2 portion of the trial follows the…
Clene is scheduled to meet with the U.S. Food and Drug Administration (FDA) in the coming months to discuss new data and a potential path toward accelerated approval for CNM-Au8, an oral therapy being developed for amyotrophic lateral sclerosis (ALS). The company plans to argue that the…
