Car mechanics wouldn’t try to learn how cars work by only studying individual components of a car or by looking at toy cars, but this is essentially how medical science has been taught over the years. They should be working with real humans, learning how existing, fully functioning, complex human creatures work. Not focusing so deeply on misfolded proteins or just one system and correcting that single system or misfold with a pill, but reshaping the whole misfolded protein(human) mess from the inside out.