First clinical evidence demonstrating that a systemically delivered small activating RNA (saRNA) can safely upregulate an endogenous target protein in human skeletal muscle in a monogenic disease. Paired muscle biopsies reveal 3.5- to 5.3-fold increases in sarcolemmal utrophin expression, accompanied by definitive tissue histopathological...
HONG KONG, March 6, 2025 /PRNewswire/ -- Akeso, Inc. (9926. HK) ("Akeso" or the "Company") is pleased to announce the completion of patient enrollment...
