Success in this role requires excellent communication, collaboration, and problem-solving skills, as well as the ability to work effectively with cross-functional teams including Analytical Sciences, Process Development, Formulation Development, Quality, Regulatory Affairs, and Research. Our revolutionary treatments for conditions like achondroplasia (the most common form of dwarfism), PKU (phenylketonuria), CLN2, a form of Batten disease, and a number of forms of MPS (mucopolysaccharidosis) offer new possibilities for patients and families who previously had few, if any, available options.