To understand neurodegenerative diseases like ALS, researchers are looking beyond individual neurons and examining tiny bridges that connect neurons to other neurons. These bridges, called tunneling nanotubes (TNTs), shuttle molecules and proteins between cells, including small structures with specific functions …
Searching for the cause of ALS, researchers look to metabolism within cells
One distinct hallmark of ALS is the dysfunction of a tiny protein called TDP-43. This protein normally lives within a cell’s nucleus, but in the case of ALS, it becomes dysfunctional, migrates out, and aggregates into the cell’s cytoplasm. The …
Discovering how motor neurons break down in ALS
In the search for the underlying mechanisms of ALS, researchers are examining the role of even the tiniest parts of neuron cells. In fact, studies have shown that in ALS patients, microtubules—which are the major component of the cell cytoskeleton …
Les Turner ALS Center at Northwestern Medicine and Sean M. Healey & AMG Center for ALS Establish Acceleration Centers of Enrollment Initiative
Together with the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, the Les Turner ALS Center at Northwestern Medicine is thrilled to announce the launch of the Acceleration Centers of Enrollment (ACE) initiative. The ACE initiative …
‘New’ ALS gene destabilizes neuron’s structure and chokes off its nucleus
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% …
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