Therapy for the mucopolysaccharidoses.

  title={Therapy for the mucopolysaccharidoses.},
  author={Vassili Valayannopoulos and Frits A. Wijburg},
  volume={50 Suppl 5},
Better understanding of disease pathophysiology, improved supportive care and availability of disease-specific treatments for some of the mucopolysaccharidosis (MPS) disorders have greatly improved the outlook for patients with MPS disorders. Optimal management of these multisystemic disorders involves a multidisciplinary team and regular, comprehensive follow-up. Enzyme replacement therapy (ERT) is now available for MPS I (Hurler, Hurler-Scheie and Scheie syndromes) (laronidase), MPS II… CONTINUE READING


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