A new drug has been approved by the Food and Drug Administration (FDA) for a rare, inherited form of amyotrophic lateral sclerosis (ALS), a paralyzing neurological disease. Known as tofersen, the drug has been shown to slow progression of the deadly disease. International clinical trials of tofersen, developed by the global biotechnology company Biogen Inc., […]
Timothy Miller, MD, PhD, the David Clayson Professor of Neurology at Washington University School of Medicine in St. Louis, has been named a winner of the Rainwater Annual Prize for Outstanding Innovation in Neurodegenerative Disease Research. The prize recognizes scientific progress that could lead to innovative, effective treatments for neurodegenerative diseases associated with the accumulation […]
“These trials don’t go without huge input and commitment from the participants and of course, their caregivers, and the clinical trial sites. I have the privilege of speaking to you about the trial as the lead author of this paper, but this is a team effort and includes a large group of people from multiple […]
An investigational drug developed to treat a rare, inherited form of amyotrophic lateral sclerosis (ALS) reduced molecular signs of the fatal, paralyzing disease and curbed neurodegeneration — but at the six-month mark, the drug did not improve motor control and muscle strength, according to results from a phase 3 clinical trial led by researchers at […]