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Research output

1. Limited data to evaluate real-world effectiveness of enzyme replacement therapy for mucopolysaccharidosis type I

   Research output: Contribution to journal › Review article › Academic › peer-review

2. Capturing MPS I: From early diagnosis to late complications

   Research output: Thesis › Phd-Thesis - Research and graduation internal

3. Failure to shorten the diagnostic delay in two ultra-orphan diseases (mucopolysaccharidosis types I and III): potential causes and implications

   Research output: Contribution to journal › Article › Academic › peer-review

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