← All indications
Mucopolysaccharidosis IV
Genetic disorder of mucopolysaccharide metabolism characterized by skeletal abnormalities, joint instability, development of cervical myelopathy, and excessive urinary keratan sulfate. There are two biochemically distinct forms, each due to a deficiency of a different enzyme.
Drugs by status
Approved for this indication (1)
Phase 2 trials (2)
Phase 1 trials (1)
Organizations
Organization Involved with Phase 2 Indications (2)