Six members of the U.S. Food and Drug Administration’s Peripheral and Central Nervous System Drugs Advisory Committee voted not to approve Amylyx Pharmaceuticals’ new drug application (NDA) for the company’s amyotrophic lateral sclerosis (ALS) candidate AMX0035, while four voted yes.
Research Roundup: Neurons Key to Parkinson’s ID’ed, New ALS Mechanism DiscoveredAmyotrophic Lateral Sclerosis (ALS), Brain Stem, Cardiovascular Disease, COVID-19 Vaccines, Diabetes, Diet, Efficacy, Frontotemporal Dementia (FTD), Gut, Immune System, Johns Hopkins, National Institutes of Health, Neurons, Obesity, Parkinson's Disease, R&D, Severe Covid-19
Researchers at the University of Copenhagen – The Faculty of Health and Medical Sciences found that activating a specialized group of neurons within the brainstem of mice restored full movement function in mice with symptoms of Parkinson’s disease.
Researchers have unlocked a key mechanism behind the genetic variant which could prove hopeful for new treatments for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).
Amylyx Pharmaceuticals Inc. announced the publication of results from the pivotal CENTAUR trial evaluating AMX0035 – an investigational neuroprotective therapy designed to reduce the death and dysfunction of motor neurons – in people with amyotrophic lateral sclerosis (ALS) in the New England Journal of Medicine.
Shares of Aquestive Therapeutics were up more than 18 percent following approval from the U.S. Food and Drug Administration for Exservan, a treatment for amyotrophic lateral sclerosis (ALS).
Novartis’ gene therapy treatment for spinal muscular atrophy could receive marketing approval as early as May 2019 after the U.S. Food and Drug Administration granted AVXS-101, now dubbed Zolgensma, priority review.
Frustrated by delays in new medicines reaching their own country, a small but growing number of patients are turning to an online broker that bills itself as a legal version of the Dallas Buyers Club.
Pfizer Inc. announced plans to invest $600 million in biotechnology and other emerging growth companies through Pfizer Ventures, the company’s venture investment vehicle.
ProMIS Neurosciences Inc., a biotechnology company focused on the discovery and development of precision treatments for neurodegenerative diseases, announced it has issued a new scientific white paper entitled: ‘Not all Amyloid Beta Therapeutics are Created Equal’.
Sangamo Therapeutics Inc. and Pfizer Inc. are partnering to develop a gene therapy to treat a disease that affects nerve cells in the brain and the spinal cord.