Enzyme Replacement Therapy in Mucopolysaccharidosis II Patients Under 1 Year of Age.

@article{Lampe2014EnzymeRT,
  title={Enzyme Replacement Therapy in Mucopolysaccharidosis II Patients Under 1 Year of Age.},
  author={Christina Lampe and Andrea Atherton and Barbara K. Burton and Maria Descartes and Roberto Giugliani and Dafne Dain Gandelman Horovitz and Sandra Obikawa Kyosen and Tatiana de S{\'a} Pacheco Carneiro Magalh{\~a}es and Ana M Martins and Nancy J. Mendelsohn and Joseph Muenzer and Laurie Denise Smith},
  journal={JIMD reports},
  year={2014},
  volume={14},
  pages={99-113}
}
Mucopolysaccharidosis (MPS) II, or Hunter syndrome, is a lysosomal storage disease characterized by multi-systemic involvement and a progressive clinical course. Enzyme replacement therapy with idursulfase has been approved in more than 50 countries worldwide; however, safety and efficacy data from clinical studies are currently only available for patients 1.4 years of age and older. Sibling case studies of infants with MPS I, II, and VI who initiated ERT in the first weeks or months of life… CONTINUE READING
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