The MPS I registry: Design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis Type I

Gregory M. Pastores, Pamela Arn, Michael Beck, Joe T.R. Clarke, Nathalie Guffon, Paige Kaplan, Joseph Muenzer, Denise Y.J. Norato, Elsa Shapiro, Janet Thomas, David Viskochil, J. Edmond Wraith

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156 Scopus citations

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