Does early use of enzyme replacement therapy alter the natural history of mucopolysaccharidosis I? Experience in three siblings.

@article{Laraway2013DoesEU,
  title={Does early use of enzyme replacement therapy alter the natural history of mucopolysaccharidosis I? Experience in three siblings.},
  author={Sarah Laraway and Catherine G Breen and Jean Mercer and Simon Jones and James Ed Wraith},
  journal={Molecular genetics and metabolism},
  year={2013},
  volume={109 3},
  pages={315-6}
}
Enzyme replacement therapy is widely used as treatment for mucopolysaccharidosis I (MPS I), and there is evidence that this produces improvement in certain clinical domains. There does appear to be variation in the response of clinical features to treatment once these are established. In a reported sibling pair, when enzyme replacement therapy was commenced pre-symptomatically in the younger child, the natural history of the condition appeared to be affected. We present data from three siblings… CONTINUE READING

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