SOD1-ALS

Health Canada is now reviewing Qalsody (tofersen) as a potential treatment for people with amyotrophic lateral sclerosis (ALS) who carry mutations in the SOD1 gene, according to the therapy’s developer Biogen. The Canadian regulatory authority agreed to review Biogen’s application for the therapy’s approval, with its decision expected in early 2025, according to…

The recent conditional approval of Qalsody (tofersen) for adults with amyotrophic lateral sclerosis (ALS) who carry mutations in the SOD1 gene is a major cause for celebration, according to the Muscular Dystrophy Association (MDA). The therapy earned accelerated approval from the U.S. Food and Drug Administration (FDA)…

Changes in neurofilament light chain (NfL) levels — a biomarker of nerve cell damage — were deemed by an advisory committee reasonably likely to predict clinical efficacy from tofersen in people with amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations. The unanimous vote from the Peripheral and Central Nervous…

The U.S. Food and Drug Administration (FDA) has agreed to review an application from Biogen for approval of the company’s experimental therapy tofersen to treat amyotrophic lateral sclerosis (ALS) associated with mutations in the SOD1 gene. The FDA has granted the application accelerated review and a decision…

Tofersen, Biogen’s investigational therapy for people with familial amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations, was safe and generally well-tolerated over three months, and appeared to lower SOD1 protein levels in the central nervous system, a Phase 1/2 clinical trial has found. There…

Voyager Therapeutics’ experimental therapy VY-SOD102 shows promise for treating patients with familial amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene, according to results in animal models. The preclinical data were presented at the Congress of the European Society of Gene and Cell Therapy…

Voyager Therapeutics presented new data from a study in an animal model of  its clinical candidate VY-SOD101, showing the therapy is effective as it targets the most common cause of familial amyotrophic lateral sclerosis (ALS). The presentation, along with another preclinical program data presentation on a Huntington’s disease therapy…