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,…
Committee favors EU approval of Qalsody for adults with SOD1-ALS
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…
Health Canada has issued a notice of noncompliance-withdrawal saying it won’t approve the oral therapy masitinib as an add-on treatment for amyotrophic lateral sclerosis (ALS). Its developer, AB Science, can submit a request for reconsideration within the next month, which it plans to do, it announced in…
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…
After a great deal of introspection, I decided for this month’s column to reflect on my life as it stands now. I wanted to write about it without being too ominous and dreadful, but I don’t think that’s possible. My love for music prompted the headline, because within the…
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…
Last Friday night, I pulled a prescription bottle out of the cupboard, preparing to take my daily antidepressant — just one half-pill of the smallest dose, but that’s been enough to take the edge off and help me cope with the ongoing grief of living with my husband’s…
An experimental gene therapy called CTx1000, which is designed to reduce toxic buildups of the protein TDP-43 in nerve cells, was found to significantly extend survival and improve motor function in mouse models of amyotrophic lateral sclerosis (ALS) and the related disorder frontotemporal dementia, known as FTD. According to…
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…