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Biohaven‘s therapy candidate verdiperstat did not significantly slow functional decline among people with amyotrophic lateral sclerosis (ALS) in the HEALEY ALS platform trial, failing to meet its primary goal, data show. Key secondary efficacy goals, such as survival and changes in lung function and muscle strength, also were…

Cytokinetics has launched its fifth annual Communications Grant Program to support patient advocacy groups working with amyotrophic lateral sclerosis (ALS) and other communities. A total of five $20,000 grants will be given to winning organizations to help them improve their outreach and patient engagement. The deadline for applications…

Treatment with a 30 mg dose of CNM-Au8 significantly reduced the risk of death, and the risk of death and permanent assisted ventilation, by more than 90% among people with amyotrophic lateral sclerosis (ALS), according to new top-line data from the CNM-Au8 arm of the HEALEY ALS platform…

Using a digital tool to assess disease severity remotely is feasible for people with amyotrophic lateral sclerosis (ALS) and may improve patient monitoring between visits to the clinic, a study suggests. The tool makes the revised ALS Functional Rating Scale (ALSFRS-R) available to patients via a computer or mobile…

Note: This story was updated Sept. 30, 2022, to correct the spelling of Albrioza and clarify the FDA advisory committee voted in March that the evidence from CENTAUR was not sufficient to support Relyvrio’s efficacy in ALS. It also added Relyvrio’s list price is set at about $158,000 per year.

There is no compelling evidence to suggest any medication increases the risk of developing amyotrophic lateral sclerosis (ALS), according to a review of published studies. If anything, classes of drugs including anti-hypertensives, cholesterol-lowering statins, aspirin, and oral contraceptives may be associated with a lower risk of ALS, although several…

Cellenkos will soon launch two Phase 1 clinical trials to investigate the safety and potential efficacy of its regulatory T-cell-based — Treg cell — therapy CK0803 for amyotrophic lateral sclerosis (ALS). The biotech’s announcement follows the approval, by the U.S. Food and Drug Administration (FDA), of its investigational…

Early treatment with CNM-Au8 continues to demonstrate survival benefits in people with amyotrophic lateral sclerosis (ALS) compared with those who started treatment nine months later, according to an update from its developer, Clene Nanomedicine. The updated results come from the RESCUE-ALS Phase 2 trial (NCT04098406) and its open-label…

The U.S. Food and Drug Administration (FDA) and the National Institutes of Health (NIH) have forged a partnership that seeks to advance both the understanding of rare neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS) and the development of potential treatments. The public-private collaboration, called the Critical Path…

Early treatment with Biogen’s experimental therapy tofersen slows disease progression in people with amyotrophic lateral sclerosis (ALS) associated with SOD1 gene mutations, compared with patients who started treatment after a six-month delay, according to findings from a Phase 3 trial and its open-label extension study. These benefits…