News

Mutations in the C9ORF7 gene, the most common genetic cause of amyotrophic lateral sclerosis (ALS), lead to defects in the stem cells needed for brain development in the early stages of life, a new study shows. In animal models of the disease, this resulted in smaller than normal volumes…

Treatment with CNM-Au8 was found to significantly delay disease progression and prolong survival, by more than 1.5 years, among adults with early-stage amyotrophic lateral sclerosis (ALS) in the RESCUE-ALS trial and its open-label extension phase. That’s according to two different statistical analyses, which overall showed that patients who…

A new two-year, $400,000 grant will support studies — by EverythingALS and the Sean M. Healey and AMG Center for ALS at Massachusetts General Hospital (MGH) — aimed at identifying early digital diagnostic markers in amyotrophic lateral sclerosis (ALS). The award, from ALS Finding a Cure…

Scientists have devised a technique for transplanting healthy motor neurons — the nerve cells that are lost in amyotrophic lateral sclerosis (ALS) — in a mouse model with “highly aggressive” disease. Transplanted motor neurons, given stimulation, were able to form healthy connections with muscle cells to control the animals’ muscle…

Akava Therapeutics will soon launch a Phase 1 clinical trial to test AKV9, its investigational therapy for amyotrophic lateral sclerosis (ALS), in healthy people. The trial follows the clearance of Akava’s investigational new drug (IND) application by the U.S. Food and Drug Administration (FDA). It will be AKV9’s…

While eating a high-calorie diet is safe and well tolerated, it does not appear to slow disease progression or reduce the risk of death in people with amyotrophic lateral sclerosis (ALS), a review of published clinical trials found. Combining data from multiple trials helps to create a single, more…

BL-001, an investigational gut-targeted therapy being developed for people with amyotrophic lateral sclerosis (ALS) and Dravet syndrome, was found to be safe and well tolerated among healthy volunteers in a Phase 1 clinical trial. Findings overall indicate that Bloom Science‘s BL-001 can be given safely to humans at…

Some $2 million in new grants from two U.S. nonprofits are seeking to help scientists globally to find ways to slash the time it takes for an amyotrophic lateral sclerosis (ALS) diagnosis. It’s now estimated to take up to 16 months from symptom onset before most individuals receive a…

People with amyotrophic lateral sclerosis (ALS) who require noninvasive ventilation in later disease stages use significantly higher opioid doses and for more extended periods than do patients who never need mechanical ventilation, according to a new study in Japan. The opioids were started for these individuals mostly to help…

Mutations in the gene NEK1, a major genetic cause of amyotrophic lateral sclerosis (ALS), may work to drive the disease by destabilizing the structure of nerve fibers and disrupting the movement of molecules inside nerve cells, according to a new study. The findings, which show for the first time how…