OrphAI Therapeutics’ experimental therapy AIT-101 has been awarded orphan drug status in the European Union for amyotrophic lateral sclerosis (ALS), following a similar designation granted in the U.S. last year. In Europe, orphan drug status is given to medicines designed to treat life-threatening or chronically debilitating conditions affecting…

Treatment with CNM-Au8 was shown to significantly prolong survival — relative to the natural history of the disease — among people with advanced amyotrophic lateral sclerosis (ALS) who took part in two expanded access programs (EAP), also known as compassionate use programs. The findings from these EAPs, which…

After years of living on the edge of life and death with my husband’s ALS, I sometimes wonder if my body has quit reacting to stress in a normal way. Trying to squeeze in lunch before I ran to town for our 18-year-old daughter’s doctor appointment, I reheated…

An advisory committee of the European Medicines Agency (EMA) has recommended that Biogen’s Qalsody (tofersen) be approved in the European Union under exceptional circumstances to treat adults with amyotrophic lateral sclerosis (ALS) associated with SOD1 gene mutations. The positive opinion from the Committee for Medicinal Products for Human Use,…

Last week, I received an email from a newly diagnosed ALS patient asking questions about rollators (walkers with wheels). “I think it’s time I begin using one,” he wrote, “but I don’t want to. What can I do instead?” I immediately felt empathy and sympathy for his…

ProJenX has been given the green light to expand its ongoing Phase 1 clinical trial of prosetin for amyotrophic lateral sclerosis (ALS) across the Atlantic Ocean to testing sites in Europe. The PRO-101 trial (NCT05279755), launched in 2022, is a three-part study evaluating prosetin’s safety, tolerability, and pharmacological…

Denali Therapeutics’ SAR443820 (DNL788) treatment candidate — now in late-stage clinical testing for amyotrophic lateral sclerosis (ALS), per the company’s website — has failed to significantly slow disease progression in adults in a Phase 2 trial. The experimental therapy “did not meet the primary endpoint of change” in outcomes…

TPN-101, an investigational oral molecule developed by Transposon Therapeutics, reduced multiple biomarkers of neuroinflammation and neurodegeneration, and slowed respiratory decline in some people with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD). That’s according to an interim analysis of a Phase 2a clinical trial (NCT04993755) that’s testing…

An unprecedented $58 million donation to the ALS Association is a “beacon of hope” for the amyotrophic lateral sclerosis (ALS) community and will finance treatment centers, aid to financially strapped patients, and clinical trials of promising treatments, according to the U.S.-based nonprofit. The donation from the estate…

Treatment with the experimental oral therapy ATH-1105 significantly reduced nerve damage, improved motor function, and prolonged survival in preclinical models of amyotrophic lateral sclerosis (ALS), a study reports. The work was funded by ATH-1105’s developer Athira Pharma, which plans to move the…