As many as 2% of all ALS cases have been linked to mutations in the gene NEK1, making it one of the top-known causes of the disease. But it wasn’t known how the mutated gene disrupts the function of the …
New results show promise in treatment of SOD1-ALS
New results from the Phase 3 clinical trial of tofersen, a drug in development by Biogen, have shown promise in treatment of superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). According to the 12-month data, earlier initiation of treatment with …
NU-9 Update: Experimental drug repairs upper motor neurons in mouse model
As connecting fibers between motor neurons, axons help deliver vital messages between the brain and the spinal cord. For people living with ALS, deteriorating axons cause that connection to break, contributing to paralysis and death. Early research on NU-9, an …
Breaking News: FDA Approves Oral Edaravone
This story has been updated with a link to the informational webinar about the new medication. The U.S. Food and Drug Administration has approved RADICAVA ORS®, an oral form of edaravone that reports the same efficacy as RADICAVA® for intravenous infusion. …
Research News from the Les Turner ALS Center at Northwestern Medicine
The 40+ research scientists at the Les Turner ALS Center at Northwestern Medicine investigate a wide range of biologic mechanisms in ALS basic research. We are pleased to announce the recent publication of three new research papers from some of …
Our Oral Testimony to the FDA Advisory Committee on the Approval of AMX0035
Open Public Hearing FDA Advisory Committee on AMX0035 March 30, 2022 Good afternoon. My name is Andrea Pauls Backman, CEO of the Les Turner ALS Foundation. My only disclosure is that the Les Turner ALS Foundation receives less than 2% …
Comments on Amylyx Pharmaceuticals’ New Drug Application for AMX0035
RE: Docket No. FDA-2018-N-0410 Dear FDA Advisory Committee: Founded in 1977, the Les Turner ALS Foundation is the oldest independent ALS group in the country. For 45 years, it has been our mission to provide the most comprehensive care and …
Response to NINDS Request for Information on ALS Strategic Plan
February 11, 2022 Dear NINDS, As we set our sights on shaping the strategic future of ALS research, we thank you for the opportunity to identify key research and priorities at NIH and beyond. We also recognize …
Faces of ALS: Working to Make True on a Wish
When Jonathan Brent, MD, PhD, asks his ALS patients what their goals are, the answer is almost always the same: to find a cure for ALS. “What motivates me,” says Dr. Brent, “is how I can make true on that …
Faces of ALS: Hope & Action in the ALS Community
“There is more hope and more action happening right now within the ALS community than I ever thought I would see in my lifetime,” says Deb Paust of Grayslake, Illinois. “It is empowering to be a part of that change.” …
