Magdalena Kegel,  —

A cell transport mechanism called endocytosis may be flawed in people with amyotrophic lateral sclerosis, a study reports. The flaw may contribute to the buildup of protein clumps in muscle-controlling motor nerve cells, a hallmark of ALS. In a study published in the journal Nature Communications, a University of…

When discussing potential Radicava (edaravone) treatment with amyotrophic lateral sclerosis (ALS) patients, physicians need to take great care in presenting facts in an ethical and scientifically sound manner, warn two researchers who believe that mainstream reports of the treatment may be misleading. The duo — Crystal Yeo, MD, PhD,…

Cell stress, such as a viral infection, may trigger the production of faulty proteins from the C9orf72 gene, a leading contributor of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), say researchers at the University of Michigan. Their study, “RAN translation at C9orf72-associated repeat expansions is selectively enhanced…

Gene editing may be a viable tool to slow disease-causing processes in amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene, a mouse study indicates. Published in the journal Science Advances, the study demonstrated that the method delayed disease…

The Wave Life Sciences therapy WVE-3972-01 reduced markers of the form of ALS that scientists believe stems from a gene mutation, studies show. Researchers said the potential treatment blocked faulty versions of messenger RNA associated with the production of abnormal C9orf72 protein. The RNA is an intermediary between the C9orf72 gene…

Using stem cell treatments to protect motor neurons is not enough to prevent the loss of nerve-muscle connections and disease progression in a mouse model of amyotrophic lateral sclerosis (ALS), researchers from Italy found. The study, published in the journal Stem Cell Research, had exposed mice to repeated brain…

The investigational gene therapy VM202, developed by VM BioPharma and intended for the treatment of amyotrophic lateral sclerosis (ALS), has been shown to be safe and well-tolerated in a small Phase 1/2 clinical trial, according to data published in the journal Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. While the study,…

Researchers at University of Exeter Medical School in the United Kingdom have started an ambitious project to investigate how mitochondria flaws may cause the neuron cell death associated with ALS. Not only may their insights provide a better understanding of how amyotrophic lateral sclerosis arises, but it also may…

Mutations in the C9ORF72 gene of people with amyotrophic lateral sclerosis (ALS) might make it more difficult for them to use brain-computer interfaces, says a study published in the journal Scientific Reports. The study, “Expansion of C9ORF72 in amyotrophic lateral sclerosis correlates with brain-computer interface performance,” suggests not only…

Researchers have discovered a new way by which a gene mutation can kill neurons in people with ALS and a related disease, frontotemporal dementia, or FTD. Their discovery, published in the journal Neuron, could offer researchers a new approach to treating these incurable conditions. The gene mutation leads to…