TAK-609 PTA Program

Hunter Syndrome or Mucopolysaccharidosis II (MPS II) is a rare genetic disorder caused by decreased levels of a specific enzyme, iduronate-2-sulfatase. Lack of this enzyme causes buildup of large sugar molecules called glycosaminoglycans (GAGs). The build up of GAGs causes significant damage to the body and brain. Idursulfase-IT, an enzyme replacement therapy, was developed for the treatment of patients with Hunter Syndrome with cognitive impairment to help with the central nervous system manifestations of the disease. The purpose of this post-trial access program is so that patients with Hunter syndrome who previously received monthly administration of idursulfase-IT through participation in an earlier clinical research study can continue to receive it.