News

Eledon’s Antibody, Tegoprubart, Shows Promise in ALS Phase 2 Trial

Eledon Pharmaceuticals’ investigational antibody-based therapy, tegoprubart, safely and effectively reduces levels of inflammatory biomarkers in adults with amyotrophic lateral sclerosis (ALS), according to top-line data from a Phase 2a clinical trial. Exploratory findings suggested that lower levels of inflammatory biomarkers and whether tegoprubart’s target is significantly reduced,…

World-renowned ALS Researcher, Clinician to Receive MDA Award

Stanley H. Appel, a world-renowned expert in amyotrophic lateral sclerosis (ALS) and the founder of the first multidisciplinary clinic dedicated to ALS care and research in the U.S., will receive the Tribute Award from the Muscular Dystrophy Association (MDA). The award acknowledges the neurologist’s work in pioneering ALS…

Gene Therapy Lowers Toxic RNA Due to C9ORF72 Mutations in ALS

One-time delivery of a CRISPR-based gene therapy significantly reduced the buildup of toxic RNA molecules resulting from C9ORF72 mutations in cells and mouse models of amyotrophic lateral sclerosis (ALS), the therapy’s developer, Locanabio, reported. The approach was designed using the company’s CORRECTx platform, which uses CRISPR technology to…

Cytokinetics Reaffirms Years-long Partnership With ALS Association

After several years of support, the late-stage biopharmaceutical company Cytokinetics is reaffirming its commitment to the ALS Association and its efforts to battle amyotrophic lateral sclerosis (ALS) and assist the ALS community. In addition to backing nationwide organizational efforts, Cytokinetics has, for more than a decade, provided support…