News

Familial ALS accounts for 8% of all cases, analysis suggests

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…

Stem cell-based therapy deemed safe in Phase 2 study

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…

Novel AI-powered platform aims to help ALS patients to communicate

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…

Coya aligns with FDA on plans for developing COYA 302 for ALS

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…

Arbor, 4DMT to develop novel gene therapies for ALS, other diseases

Arbor Biotechnologies and 4DMT — two U.S. companies working in next-generation genetic medicines — have established a strategic partnership to develop and commercialize novel gene therapies for neurological diseases, including amyotrophic lateral sclerosis (ALS). The agreement includes the codevelopment of up to six product candidates for diseases…

NurOwn and its exosomes given patents in Europe, Australia, Israel

BrainStorm Cell Therapeutics has received patents in Europe, Australia, and Israel covering the use of investigational NurOwn and NurOwn exosomes in treating amyotrophic lateral sclerosis (ALS) and other neurological disorders. The European patent specifically covers the use of an isolated population of mesenchymal stem cells —…

Juvenile-onset ALS linked to new SPTLC2 mutation: Study

A previously unknown mutation in the SPTLC2 gene was identified in two patients who developed juvenile-onset amyotrophic lateral sclerosis (ALS), a study reports. The mutation significantly increased the production of certain types of fat-like molecules called sphingolipids, resulting in early-onset muscle weakness, progressive motor impairment, and involuntary tongue movements.