Hurler syndrome gene therapy shows early success in a small study
Hurler syndrome (also called mucopolysaccharidosis type 1, Hurler variant [MPSIH]), is a genetic disorder that causes abnormal musculoskeletal development,...
hematopoietic stem cell
Hurler syndrome (also called mucopolysaccharidosis type 1, Hurler variant [MPSIH]), is a genetic disorder that causes abnormal musculoskeletal development,...