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Blocking certain inflammatory signaling pathways may be one way to reverse nerve cell alterations that drive the symptoms of amyotrophic lateral sclerosis (ALS), according to preclinical research using a mouse model of the disease. In the mice, an ALS-associated mutation in the FUS gene caused caused motor neurons — the…

Up to three years of daily treatment with the investigational therapy IPL344 was well tolerated and slowed disease progression among adults with amyotrophic lateral sclerosis (ALS), according to top-line data from a small Phase 1/2a clinical trial. Preliminary efficacy analyses also indicated that IPL344 might be able to…

Note: This story was updated Jan 24, 2024, to correct that Radicava and Radicava Oral Suspension are available in Switzerland but not in other European countries. FAB122, an oral formulation of edaravone developed by Ferrer, failed to slow disease progression or extend survival in adults with amyotrophic lateral…

The proportion of people diagnosed with familial amyotrophic lateral sclerosis (ALS) among all cases is 8%, according to a pooled analysis of studies published worldwide. Still, data show there was a wide variation of estimates across the studies based partly on geographical differences, study design, the definition of familial…

CL2020, a stem cell-based therapy that was being developed by the Life Science Institute, part of Mitsubishi Chemical, was found to be safe and  tolerated well by five people with amyotrophic lateral sclerosis (ALS) who took part in a Phase 2 clinical study. The investigational therapy, which involved…

An initiative at a University of Illinois campus that aims to improve voice recognition software for people with speech difficulties is recruiting adults in the U.S. and Puerto Rico living with amyotrophic lateral sclerosis (ALS). Known as the Speech Accessibility Project, it is led by the Urbana-Champaign…

Verge Genomics has launched a proof-of-concept trial to test whether VRG50635, its oral PIKfyve inhibitor therapy, can be used as a potential treatment for people with amyotrophic lateral sclerosis (ALS). The Phase 1b study will assess the safety and tolerability of escalating doses of VRG50635 — which works…

Researchers have unveiled a new AI-powered avatar platform — one making use of artificial intelligence (AI) technology — that aims to help people with amyotrophic lateral sclerosis (ALS) to communicate, even when the neurodegenerative disease progresses to the point that speaking and moving become difficult or impossible. The platform…

After two meetings with the U.S. Food and Drug Administration (FDA), Coya Therapeutics says it’s received constructive feedback — and has reached an alignment with the agency regarding development plans for COYA 302, its therapy candidate for people with amyotrophic lateral sclerosis (ALS). The company held the two…