Research output

  1. Patients’ view on gene therapy development for lysosomal storage disorders: a qualitative study

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

  2. Early start of enzyme replacement therapy in pediatric male patients with classical Fabry disease is associated with attenuated disease progression

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

  3. A review of treatment modalities in gyrate atrophy of the choroid and retina (GACR)

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

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ID: 44862