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,…
News
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…
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…
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…
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…
FundaMental Pharma’s investigational molecule FP802 reduced neurological disability, protected nerve cells, and prolonged survival in a mouse model of amyotrophic lateral sclerosis (ALS), according to recent research. The molecule also protected tissue from glutamate toxicity, a type of cellular toxicity that’s implicated…
Mutations in the C9ORF72 gene may lead to amyotrophic lateral sclerosis (ALS) and the related disorder frontotemporal dementia (FTD) in part by boosting the production of a powerful inflammatory protein called interleukin 17A (IL-17A), recent preclinical research suggests. Inhibiting IL-17A via genetic manipulations or…
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