ResearchRSSMay 15
RNA therapy shows promise for addressing key driver of ALS
Scientists have created a new medicine made from RNA (a molecule similar to DNA) that may help reduce clumping of a protein called TDP-43 in people with ALS. ALS is a disease that affects nerve cells and causes muscle weakness. This new therapy works by stopping the protein from clumping together, which is a major problem in ALS. The research team studied exactly how this medicine works at the molecular level.
WHY IT MATTERSThis research targets TDP-43 clumping, which is a core driver of ALS neurodegeneration, potentially offering a disease-modifying approach for patients who currently have limited treatment options.