PTC-124(10毫克)产品是PTC-124 has been reported to help the ribosome skip over the premature stop, restore production of functional protein, and thereby potentially treat these genetic diseases. In a mouse model of cystic fibrosis caused by nonsense mutations, PTC-124 treatment has been shown to restore cystic fibrosis transmembrane conductance regulator (CFTR) protein expression and function.