Information for the public
The condition and the treatment
Mucopolysaccharidosis type IVa (MPS IVa), or Morquio syndrome, is a very rare, inherited condition. It is caused by a missing enzyme that the body needs for connective tissue growth. It causes a wide range of symptoms that get worse over time, including breathing problems, joint and skeletal abnormalities, hearing loss, problems with eye sight and heart valve disease.
The aim of treatment with elosulfase alfa (Vimizim) is to replace the missing enzyme.