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…
Blocking channels containing the connexin 43 (Cx43) protein on astrocytes — a type of nerve support cell — slowed disease progression in a mouse model of amyotrophic lateral sclerosis (ALS), a study found. The protein was found to be increased in ALS patient tissues and spinal fluid, and its…
A first healthy volunteer has been enrolled in a Phase 1 trial and given a first dose of PrimeC, an investigational combination therapy for people with amyotrophic lateral sclerosis (ALS), the treatment’s developer, NeuroSense Therapeutics, announced. The open-label Phase 1 trial (NCT05232461) is a pharmacokinetic study,…
ALPHA-0602, an investigational gene therapy for amyotrophic lateral sclerosis (ALS), can successfully increase progranulin levels and reduce the toxicity of TDP-43 protein aggregates in cell cultures and mouse models, Alpha Cognition, the therapy’s developer, announced. “These new insights from our preclinical research further support the development of ALPHA-0602…
Team Drea Foundation is launching a customizable wellness challenge that seeks to raise $50,000 in 50 days for amyotrophic lateral sclerosis (ALS) research. The 50for50 challenge invites people to set a personal goal related to the number 50, such as 50 pushups a day or 50 acts…
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