TDP-43 clumps

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…

Doing a muscle biopsy to look for abnormal clumps of the TDP-43 protein in the nerve fibers of muscle tissue may be useful for the early detection of amyotrophic lateral sclerosis (ALS), according to a single-center study from Japan. “It is difficult to diagnose ALS in its early stages…

Maze Therapeutics is advancing the development of a lead gene therapy candidate for amyotrophic lateral sclerosis (ALS), which works by suppressing the activity of a potent genetic modifier called ATXN2. Genetic modifiers are genes or genetic variants that can increase or reduce the severity of a condition without necessarily…

Pharnext’s investigational oral combination therapy PXT864 prevents degeneration of motor neurons and their point of contact with muscle cells, as well as the buildup of toxic TDP-43 protein aggregates — all hallmarks of amyotrophic lateral sclerosis (ALS) — in cellular models of the disease. The data…

A cancer treatment called tamoxifen, which previously showed promise in preclinical studies for the treatment of amyotrophic lateral sclerosis (ALS), provides only modest and short-lasting benefits to patients with the disease, a small Phase 1/2 clinical trial has found. According to the study’s researchers, larger studies and longer…

ProMIS Neurosciences announced its program for amyotrophic lateral sclerosis (ALS) is advancing, and scientists were able to generate a specific type of antibody against toxic TDP-43 protein clumps — a hallmark of this disease — that continuously damage nerve cells. These antibodies, called intrabodies because they…

Target ALS and The ALS Association will jointly fund two projects focused on the development of new tests to evaluate TDP-43’s potential as a biomarker of amyotrophic lateral sclerosis (ALS). Nearly all ALS cases are characterized by the formation of toxic aggregates (clumps) containing the TDP-43 protein. These…