AUT00201, an experimental oral small molecule being developed by Autifony Therapeutics, reduced the loss of motor neurons — the nerve cells that control movement — and eased motor symptoms in a mouse model of amyotrophic lateral sclerosis (ALS), a new study showed. It was also found to improve…
China’s Center for Drug Evaluation (CDE) has approved a request by Zhimeng Biopharma to launch a Phase 2/3 study of its experimental therapy CB03-154 in people with amyotrophic lateral sclerosis (ALS). The oral treatment, which is designed to slow disease progression by reducing nerve cell overactivity, is also…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Klotho Neurosciences’ gene therapy candidate for amyotrophic lateral sclerosis (ALS). The designation is intended to boost the development of potential treatments for rare diseases, or those that impact fewer than 200,000 people in the U.S. It…
A new treatment approach that noninvasively delivers gentle electrical stimulation to the spinal cord and peripheral nerves — the nerves that connect the brain and spinal cord to the rest of the body — was found to slow disease progression and extend survival in a mouse model of amyotrophic…
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 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…
Klotho Neurosciences is advancing KLTO‑202, its gene therapy candidate for amyotrophic lateral sclerosis (ALS), to the manufacturing and process development phase — with an eye toward trials in humans beginning next year. The manufacturing phase is expected to last about eight months, and will be followed by 4-6…
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 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…
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…
