An experimental gene therapy called CTx1000, which is designed to reduce toxic buildups of the protein TDP-43 in nerve cells, was found to significantly extend survival and improve motor function in mouse models of amyotrophic lateral sclerosis (ALS) and the related disorder frontotemporal dementia, known as FTD. According to…
Experimental gene therapy CTx1000 extends survival in mice
TPN-101, an investigational oral molecule developed by Transposon Therapeutics, reduced multiple biomarkers of neuroinflammation and neurodegeneration, and slowed respiratory decline in some people with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD). That’s according to an interim analysis of a Phase 2a clinical trial (NCT04993755) that’s testing…
An unprecedented $58 million donation to the ALS Association is a “beacon of hope” for the amyotrophic lateral sclerosis (ALS) community and will finance treatment centers, aid to financially strapped patients, and clinical trials of promising treatments, according to the U.S.-based nonprofit. The donation from the estate…
Treatment with the experimental oral therapy ATH-1105 significantly reduced nerve damage, improved motor function, and prolonged survival in preclinical models of amyotrophic lateral sclerosis (ALS), a study reports. The work was funded by ATH-1105’s developer Athira Pharma, which plans to move the…
I’ve written before about how my late husband, Jeff, drew inspiration from Lou Gehrig, even before his ALS diagnosis in 2018. Today, I’m sharing how I now do the same through Live Like Lou, the ALS organization that bears Gehrig’s name. When I met Jeff,…
FundaMental Pharma’s investigational molecule FP802 reduced neurological disability, protected nerve cells, and prolonged survival in a mouse model of amyotrophic lateral sclerosis (ALS), according to recent research. The molecule also protected tissue from glutamate toxicity, a type of cellular toxicity that’s implicated…
Since the point where my husband, Todd, needed a wheelchair because of his ALS progression, he’s had to navigate a less than perfectly accessible world. Todd is particularly challenged in our old community, which was built up long before the passage of the Americans with Disabilities Act in…
Mutations in the C9ORF72 gene may lead to amyotrophic lateral sclerosis (ALS) and the related disorder frontotemporal dementia (FTD) in part by boosting the production of a powerful inflammatory protein called interleukin 17A (IL-17A), recent preclinical research suggests. Inhibiting IL-17A via genetic manipulations or…
Myrobalan Therapeutics will use a $400,000 grant from the ALS Association — along with $24 million in new financing — to help advance the development of its oral CSF1 receptor (CSF-1R) inhibitor program, intended to reduce neuroinflammation and promote nerve repair in people with amyotrophic lateral sclerosis…
My introduction to ALS followed the same path as many other patients. After months of worry, I was relieved to finally receive a diagnosis. But that was the tip of the iceberg. I then had to understand what ALS was and how to cope with my symptoms,…
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