News

Treatment with the investigational oral therapy masitinib can extend lifespan when given early in the course of amyotrophic lateral sclerosis (ALS), according to a new study. “These long-term survival data, with an average follow-up of 75 months since diagnosis, suggest that masitinib can offer a substantial survival benefit…

AB Science’s proposed measures to ensure patient safety in clinical trials of its experimental oral therapy masitinib were deemed sufficient by the French National Agency for Medicines and Health Products Safety (ANSM), and patient enrollment in the country may resume. The announcement comes less than two months after…

In keeping with its commitment to turn amyotrophic lateral sclerosis (ALS) into a “livable disease” by 2030, and to ramp up discovery and funding of new therapy candidates, the ALS Association is moving to a unified structure from a federated one. “Our best opportunity to fulfill this promise and…

Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept. 10…

Verge Genomics and Eli Lilly have entered into a three-year collaboration to find and develop therapies for amyotrophic lateral sclerosis (ALS). Verge will focus on discovering and validating potential new therapeutic targets, while Lilly will select up to four candidates to advance through clinical testing with a goal of…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Prilenia’s pridopidine for the treatment of amyotrophic lateral sclerosis (ALS). The decision follows a recent positive opinion from a branch of the European Medicines Agency recommending the treatment be given orphan drug status in…

Ubiquitination — the cell’s process of tagging proteins, often to degrade unwanted ones — is essential for the disassembly of stress granules that, by not being cleared, damage cells in amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, a study showed. The study, “Ubiquitination is essential for…

Coya Therapeutics’ ALS001, a potential regulatory T-cell (Treg) therapy designed to halt amyotrophic lateral sclerosis (ALS) progression, has been granted an orphan drug designation by the U.S. Food and Drug Administration (FDA). Furthermore, a Phase 2a trial (NCT04055623) testing ALS001 versus a placebo in 12 ALS patients…

Using motor neurons derived from people with amyotrophic lateral sclerosis (ALS) is a promising approach to screen for molecules that prevent certain disease mechanisms, and to identify new targets for therapies, according to a recent study. Two classes of therapies were identified that might lessen the excessive firing of motor…

Strenuous, intense exercise done frequently is an environmental risk factor for amyotrophic lateral sclerosis in people with ALS risk genes, particularly those who carry a faulty C9ORF72 gene, a study concluded. Future research is needed to better understand and identify people at disease risk due to such exercise,…