The Robert Packard Center for ALS Research at Johns Hopkins and ALS United will fund a preclinical study investigating how TDP-43 abnormalities contribute to amyotrophic lateral sclerosis (ALS). The project, “Alternative Polyadenylation-Driven Subcellular RNA Mislocalization in TDP-43 Proteinopathies,” will explore the molecular mechanisms by which TDP-43 clumps,…
Researchers have discovered a pathway in cells that prevents the formation of TDP-43 protein clumps, which contribute to nerve cell damage in nearly all people with amyotrophic lateral sclerosis (ALS). The findings may aid ALS therapies that can activate the pathway and potentially limit TDP-43 clumping and prevent nerve…
Edaravone, an approved amyotrophic lateral sclerosis (ALS) therapy sold as Radicava and Radicava ORS, may exert neuroprotective effects by correcting a key molecular feature of ALS, a cell-based study shows. Specifically, edaravone was found to correct the abnormal localization of TDP-43, a protein that is involved in many…
Neuropeutics has partnered with LifeArc to advance work on a small molecule therapy against TDP-43 clumping as a potential disease-modifying treatment for amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND). Studies in cellular and animal models found the candidate treatment,…
Bravyl (oral fasudil), an experimental therapy Woolsey Pharmaceuticals is developing for amyotrophic lateral sclerosis (ALS), significantly reduced the spread of toxic TDP-43 protein clumps in a cell model of the disease. New data was presented at the University of Denver during the International Symposium on ALS/MND, which took…
Researchers have developed a type of gene therapy that can rescue the function of the TDP-43 protein in diseased nerve cells in amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases while leaving healthy cells untouched. The approach uses problematic sequences called cryptic exons that are only present in cells…
The Swiss biotechnology company Mabylon has received two research grants from Target ALS and the ALS Association to support the development of human-derived antibodies that may help restore normal TDP-43 function in people with amyotrophic lateral sclerosis (ALS). In nearly all ALS patients, nerve cells produce…
Dewpoint Therapeutics has won a second grant from Target ALS Foundation to advance preclinical studies of its experimental TDP-43-targeted therapy for amyotrophic lateral sclerosis (ALS). TDP-43 is a protein that often gets mislocalized within cells in ALS and forms into toxic clumps that contribute to neurodegeneration. Dewpoint’s…
Reduced activity of the TDP-43 protein, a hallmark of amyotrophic lateral sclerosis (ALS), leads to changes in the DNA of nerve cells, which alters the activity of important genes, a new study reports. These findings may help explain how problems with this protein can contribute to the death of…
Dewpoint Therapeutics has received a grant from the Target ALS Foundation to advance the development of small molecules called c-mods, which will target the TDP-43 condensates thought to drive most cases of amyotrophic lateral sclerosis (ALS). Condensates are borderless compartments in cells that can cause cellular processes…
