Marisa Wexler, MS,  senior science writer—

The anticonvulsant ezogabine can significantly reduce abnormal motor neuron excitability in people with amyotrophic lateral sclerosis (ALS), a clinical trial has found. The findings support further investigation of ezogabine, and into the possible utility of neuron excitability as a measurement of disease in ALS, researchers…

The U.S. Senate has passed a bill that may make it easier for people with amyotrophic lateral sclerosis (ALS) to access disability benefits. The proposed legislation, the ALS Disability Insurance Access Act of 2019, still must be approved by the U.S. House of Representatives. If enacted, the legislation…

A lightweight, wearable sensor is being developed that can detect minute facial movements and translate them into messages, potentially helping people with amyotrophic lateral sclerosis (ALS) communicate more easily. Once produced in mass, each device is also expected to cost about $10. The sensor is described in the study…

Transplanting a combination of two types of modified bone marrow cells eased motor deficits and prolonged survival in a mouse model of amyotrophic lateral sclerosis (ALS), researchers reported. Their study, “Enhancing the therapeutic efficacy of bone marrow-derived mononuclear cells with growth factor-expressing mesenchymal stem cells for ALS…

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases.  “The RareLaunch program is…

Toxic clumps due to the buildup of the TDP-43 protein can trigger the neuronal inflammation that precedes symptoms of amyotrophic lateral sclerosis (ALS) by activating an inflammatory signaling pathway called cGAS-STING, a study reported. Its findings suggest that targeting this inflammatory signaling may be an effective way of treating ALS.

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

A $2 million grant from the National Institutes of Health (NIH) is funding work to better understand how molecular structures called protein-RNA condensates form and are regulated within cells. Findings from the five-year project could help in understanding and treating diseases where these molecular structures play a role, including…

An impairment in the activity of the RNA exosome complex, which cells use to degrade defective RNA molecules, could contribute to amyotrophic lateral sclerosis (ALS) caused by mutations in the C9orf72 gene, a new study indicates. The study, “The RNA exosome complex degrades expanded hexanucleotide…

Newly developed compounds derived from ebselen could be used to treat some types of amyotrophic lateral sclerosis (ALS), a recent study suggests. The study, “Novel Selenium-based compounds with therapeutic potential for SOD1-linked amyotrophic lateral sclerosis,” was published in EBioMedicine. About a fifth of familial…