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When the TDP-43 protein is less able to form two-protein complexes called dimers, it appears to be more likely to form the toxic clumps seen in amyotrophic lateral sclerosis (ALS), a study reports. As these clumps are found in about 97% of all ALS cases, a better understanding of the…

Note: This story was updated Sept. 25, 2023, to correct that TW001 uses the same active ingredient as Radicava and Radicava ORS but is a different therapy. Treeway has chosen Specialised Therapeutics Asia, known as ST, as its partner to market TW001, an oral formulation of edaravone, for…

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…