BioBlast Pharma, based in Tel Aviv, Israel, has been given clearance from the U.S. Food and Drug Administration (FDA) to test its experimental drug Cabaletta in the U.S. in people with oculopharyngeal muscular dystrophy (OPMD). Cabaletta is a chemical chaperone, designed to prevent abnormal protein aggregation, a phenomenon thought to contribute to muscle weakness in OPMD patients. The ultimate goal of Cabaletta treatment is to sustain or improve function, particularly in the muscles around the eyes and the swallowing muscles, which are the most affected in OPMD.
