News

Columbia gets $15M to design gene therapies for rare ALS forms

Columbia University researchers will use a $15 million federal grant to design individualized gene therapies for nine people with ultra-rare genetic forms of amyotrophic lateral sclerosis (ALS). The three-year grant comes from the National Institute of Neurological Disorders and Stroke’s (NINDS) Ultra-rare Gene-based Therapy (URGenT) Network, a program…

ASHA-624 to be studied as potential disease-modifying therapy

ASHA-624, an experimental therapy that blocks the activity of the SARM1 protein, will be developed as a potential disease-modifying therapy for amyotrophic lateral sclerosis (ALS). Developer Asha Therapeutics said it will conduct preclinical studies aimed at supporting the treatment’s potential for testing in human clinical trials. “As a veteran CNS…

Relyvrio withdrawn from US, Canada after Phase 3 trial results

Relyvrio (sodium phenylbutyrate and taurursodiol), an approved treatment for amyotrophic lateral sclerosis (ALS), was voluntarily removed from the U.S. and Canadian markets. Amylyx Pharmaceuticals, the therapy’s developer, made the decision after top-line results from the Phase 3 PHOENIX trial (NCT05021536) showed that patients who received Relyvrio…