Usefulness of bone marrow transplantation in the Hurler syndrome

Am J Cardiol. 2003 Oct 1;92(7):882-6. doi: 10.1016/s0002-9149(03)00909-3.

Abstract

The Hurler syndrome, an autosomal recessive storage disease of childhood, leads to death within the first decade of life from progressive deposition of glycosaminoglycans within the myointima of the coronary arteries and airways. Cardiac ultrasound findings of patients with this syndrome >10 years after successful bone marrow transplantation are described.

Publication types

  • Clinical Trial

MeSH terms

  • Adolescent
  • Bone Marrow Transplantation* / adverse effects
  • Cardiovascular Physiological Phenomena
  • Child
  • Female
  • Follow-Up Studies
  • Heart Diseases / diagnostic imaging
  • Heart Diseases / etiology
  • Humans
  • Male
  • Mucopolysaccharidosis I / complications
  • Mucopolysaccharidosis I / physiopathology
  • Mucopolysaccharidosis I / therapy*
  • Outcome Assessment, Health Care
  • Time
  • Ultrasonography