A protein called NOVA1 may be involved in the early disruption of protein production in amyotrophic lateral sclerosis (ALS), even before the characteristic buildup of the toxic TDP-43 protein occurs. The finding is a step toward identifying ways to treat the disease in its earlier stages, before nerve cells…
Amylyx Pharmaceuticals has partnered with Sunnybrook Research Institute (SRI) to find new therapeutic candidates for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. Under terms of the two-year sponsored research agreement, SRI will work to identify potential therapeutics that potently and specifically inhibit Bax and Bak, two proteins…
A U.S. Food and Drug Administration (FDA) advisory committee will meet  Sept. 7 to discuss the approval of AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS), Amylyx Pharmaceuticals announced. While the application was granted priority review earlier this year, with a final decision expected by June…
An at-home music therapy program aimed at improving speech, swallowing, and respiration in people with amyotrophic lateral sclerosis (ALS) was well-tolerated and feasible, according to a small pilot study. While the pilot study (NCT03604822) was not designed to test the program’s effectiveness, collected data suggest that the music…
The U.S. Food and Drug Administration has granted orphan drug designation to Tranquis Therapeutics‘ experimental therapy TQS-168 for treating amyotrophic lateral sclerosis (ALS). Orphan drug status is given to experimental therapies with promise for treating rare diseases — those affecting fewer than 200,000 people in the U.S.
The estimated 2017 U.S. prevalence of individuals with amyotrophic lateral sclerosis (ALS) was 24,821, or 7.7 per 100,000 people, according to a recent report from the National ALS Registry. While a statistical model was used to account for potentially missing data, the research team emphasized that this number is…
A research team has developed a new way to search for genes and gene mutations that may contribute to the development of rare diseases, bypassing some of the challenges typically associated with genetic analyses. For diseases like amyotrophic lateral sclerosis (ALS) in which genetic factors are thought to…
Merit E. Cudkowicz, MD,  is the winner of the inaugural Target ALS Rebecca Luker Courage Award in recognition of her work and efforts to help  amyotrophic lateral sclerosis (ALS) patients and the ALS community. Cudkowicz is the chief of neurology at Massachusetts General Hospital, a professor…
Note: Frontiers in Genetics, the publisher of the study discussed in this ALS News Today news story, received a request from the article’s authors to retract it in light of new information indicating that the variants reported in the study might not be meaningful; instead, they may actually result from…
Occupational exposure to lead — a heavy metal — prior to disease onset is associated with a more severe disease progression and lower survival among people with amyotrophic lateral sclerosis (ALS), according to a recent study. “In survival analysis of an international cohort [group] of ALS cases, our results…
