One-time delivery of a CRISPR-based gene therapy significantly reduced the buildup of toxic RNA molecules resulting from C9ORF72 mutations in cells and mouse models of amyotrophic lateral sclerosis (ALS), the therapy’s developer, Locanabio, reported. The approach was designed using the company’s CORRECTx platform, which uses CRISPR technology to…
The Jane Calmes ALS Scholarship Fund wants to help support students who wish to pursue undergraduate studies, but whose families have been impacted financially by amyotrophic lateral sclerosis (ALS). This is the fourth year The ALS Association, together with Mark Calmes, is offering support to U.S. post-high…
NeuroSense Therapeutics will soon begin a Phase 1 clinical trial to investigate the impact of food on PrimeC, a combo medication being developed as a treatment for amyotrophic lateral sclerosis (ALS), in a group of healthy volunteers. The open-label Phase 1 trial (NCT05232461) is expected to enroll…
An investigational antisense oligonucleotide (ASO) molecule that works by increasing the production of Stathmin-2 protein effectively reversed a number of neurodegenerative processes in lab-grown motor neurons, the cells that are damaged in amyotrophic lateral sclerosis (ALS), a study found. The rescue occurred even in the absence of TDP-43, a…
Repeat injections of mesenchymal stem cells (MSCs) into the spinal canal were safe and well tolerated in patients with amyotrophic lateral sclerosis (ALS), and showed potential to significantly slow the rate of disease progression, a small Phase 2 clinical trial found. “Larger studies are warranted to confirm our observations…
TQS-168, an investigational therapy being developed by Tranquis Therapeutics, reduced inflammation in mouse and human models of amyotrophic lateral sclerosis (ALS), and prolonged survival in the mice, according to a preclinical study. Based on these findings, Tranquis is moving the therapy into Phase 1 clinical studies. “We believe…
Certain blood biomarkers of lipid, or fat, metabolism — typically measured to determine a person’s risk of cardiovascular disease — appear to protect against developing amyotrophic lateral sclerosis (ALS), a study of more than 500,000 adults reported. Specifically, researchers found that people with higher levels of high density lipoprotein (HDL)…
A technology based on artificial intelligence is helping to spot biomarkers and document the progression of amyotrophic lateral sclerosis (ALS) in a large speech study being conducted by EverythingALS. The technology, developed by Modality.ai, is a web-based computer program that uses audio (speech) and video (facial) recordings…
A Temple University research team received a National Institutes of Health (NIH) grant totaling $1.25 million to explore the molecular underpinnings of neuronal degeneration in amyotrophic lateral sclerosis (ALS) and other diseases. With this five-year grant, the team led by Gareth Thomas, PhD, an associate professor of neural sciences…
Today, the National ALS Registry is joining the Major League Baseball to celebrate Lou Gehrig’s life and legacy and to raise greater awareness of amyotrophic lateral sclerosis (ALS). This year’s inaugural “Lou Gehrig Day” on June 2 honors the famous New York Yankees baseball player whose career…
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