The U.S. Food and Drug Administration (FDA) and the National Institutes of Health (NIH) have forged a partnership that seeks to advance both the understanding of rare neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS) and the development of potential treatments. The public-private collaboration, called the Critical Path…
Early treatment with Biogen’s experimental therapy tofersen slows disease progression in people with amyotrophic lateral sclerosis (ALS) associated with SOD1 gene mutations, compared with patients who started treatment after a six-month delay, according to findings from a Phase 3 trial and its open-label extension study. These benefits…
Researchers at Brigham and Women’s Hospital (BWH) have received a grant from the ALS Association to study foralumab nasal spray as a potential treatment for amyotrophic lateral sclerosis (ALS). The Lawrence & Isabel Barnett Drug Development Program grant was awarded to the Ann Romney Center for Neurologic…
The treatment development company Verge Genomics — known for its all-in-human artificial intelligence platform — has been named among the “Fierce 15” by daily email newsletter Fierce Biotech. Verge’s inclusion by the web resource in the 20th annual Fierce 15 listing, announced in a company press release,…
PrimeC, an experimental combination treatment for amyotrophic lateral sclerosis (ALS) being developed by NeuroSense Therapeutics, was well tolerated and lowered disease biomarkers in an open-label Phase 2 clinical trial. That’s according to the study, “Combination of ciprofloxacin/celecoxib as a novel therapeutic strategy for ALS,” published…
RT001, an investigational treatment for amyotrophic lateral sclerosis (ALS), shows promising signs that it can slow disease progression, particularly among patients with more severe disease, according to data from a completed Phase 2 pilot trial. While these findings indicate improvements in patients on RT001 treatment, statistical significance was…
Amylyx Pharmaceuticals has donated data from the CENTAUR Phase 2 trial to the PRO-ACT database — the largest collection ever compiled of de-identified amyotrophic lateral sclerosis (ALS) patient records from clinical trials. CENTAUR (NCT03127514) evaluated the safety and efficacy of Amylyx’s investigational treatment AMX0035 against a placebo…
Kulicke & Soffa Industries has announced a multi-year donation that ultimately will increase amyotrophic lateral sclerosis (ALS) patients’ access to potentially life-saving treatments. The contribution, for which an amount was not specified, will establish and support the multicenter expanded access protocol (EAP) companion program to the HEALEY ALS…
AMX0035 — Amylyx Pharmaceuticals’ experimental combination therapy for amyotrophic lateral sclerosis (ALS) now under review for approval in the U.S. — alters the activity of genes involved in several disease-relevant pathways, according to a study in lab-grown cells. Notably, the two-compound oral treatment was seen to change the activity of many…
The levels of five small RNA molecules, called microRNAs, inside tiny vesicles in the blood of people with amyotrophic lateral sclerosis (ALS) differ significantly from those of people without ALS and may offer a way of accurately and quickly diagnosing the disease, a study suggests. Importantly, its researchers evaluated…
