News

Advocates ask for new FDA review of stem cell therapy NurOwn

A coalition of amyotrophic lateral sclerosis (ALS) patients and their family members has filed a petition to the U.S. Food and Drug Administration (FDA) asking the agency to review again available data for the stem cell therapy NurOwn (debamestrocel). The petitioners include ALS patients who received NurOwn in…

Neurizon, Elanco sign deal to advance ALS therapy NUZ-001

Neurizon Therapeutics has entered into a global licensing deal with Elanco Animal Health to advance the development of NUZ-001, an investigational therapy for amyotrophic lateral sclerosis (ALS). Monepantel, the active ingredient in NUZ-001, is an oral deworming agent commonly used in veterinary medicine, and Elanco, a company that…

FDA grants orphan drug status to ALS gene therapy SNUG01

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Sineugene Therapeutics‘ SNUG01, a gene therapy for people with amyotrophic lateral sclerosis (ALS). The designation is given to potential treatments for rare diseases, or those affecting fewer than 200,000 people in the U.S. It provides…

Clene working with FDA on accelerated approval for CNM-Au8

Clene has incorporated suggestions from the U.S. Food and Drug Administration (FDA) on its analysis plans for certain biomarker data that’s expected to support an accelerated approval application for CNM-Au8 to treat amyotrophic lateral sclerosis (ALS), the company has announced. Specifically, the FDA had suggested revisions to…

An author discusses ALS and how she became an activist

Mindy Uhrlaub, an author and activist for amyotrophic lateral sclerosis (ALS), has spent a good part of her life caring for loved ones. Soon after she became a mom in 2003, her husband learned he had cancer. “Right as my older son was taking his first wobbly steps, my…