Lindsey Shapiro, PhD,  science writer—

Treatment with a 30 mg dose of CNM-Au8 significantly reduced the risk of death, and the risk of death and permanent assisted ventilation, by more than 90% among people with amyotrophic lateral sclerosis (ALS), according to new top-line data from the CNM-Au8 arm of the HEALEY ALS platform…

Note: This story was updated Sept. 30, 2022, to correct the spelling of Albrioza and clarify the FDA advisory committee voted in March that the evidence from CENTAUR was not sufficient to support Relyvrio’s efficacy in ALS. It also added Relyvrio’s list price is set at about $158,000 per year.

Early treatment with CNM-Au8 continues to demonstrate survival benefits in people with amyotrophic lateral sclerosis (ALS) compared with those who started treatment nine months later, according to an update from its developer, Clene Nanomedicine. The updated results come from the RESCUE-ALS Phase 2 trial (NCT04098406) and its open-label…

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…

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…

Repeated muscle injections with Engensis (VM202), Helixmith’s investigational non-viral gene therapy, were generally safe and well-tolerated in people with amyotrophic lateral sclerosis (ALS), according to top-line data from a Phase 2a clinical trial. While the sample size was too small to determine the therapy’s efficacy, muscle biopsies were…

Whole-genome sequencing, a type of genetic screening that examines a person’s complete DNA sequence, may be a promising way to identify disease-causing mutations in people with amyotrophic lateral sclerosis (ALS), according to a study of Italian patients. Disease-associated mutations were found in more than a quarter of 1,043 Italian…

Amydis is developing a test to monitor the buildup of TDP-43 in the eye’s retina of people with amyotrophic lateral sclerosis (ALS), which could offer a noninvasive way to diagnose the neurodegenerative disease sooner and better monitor its progression. The research will be conducted with support from a…

A number of stem cell lines developed to better understand how certain genetic factors might contribute to amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases is now available to scientists worldwide. The cell lines were engineered by researchers at the National Institutes of Health Center for Alzheimer’s and Related…