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Treatment Beliefs Reflect Unmet Clinical Needs in Lysosomal Storage Diseases: An Opportunity for a Patient-Centered Approach
Corazolla, E. M., Langeveld, M., Brands, M. M. M. G., Sjouke, B. & Hollak, C. E. M., 1 Mar 2025, In: JIMD reports. 66, 2, e70003.Research output: Contribution to journal › Article* › Academic › peer-review
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Different diseases, different needs: Patient preferences for gene therapy in lysosomal storage disorders, a probabilistic threshold technique survey
Corazolla, E. M., Eskes, E. C. B., Veldwijk, J., Brands, M. M. M. G., Dekker, H., van de Mheen, E., Langeveld, M., Hollak, C. E. M. & Sjouke, B., 1 Dec 2024, In: Orphanet journal of rare diseases. 19, 1, 367.Research output: Contribution to journal › Article* › Academic › peer-review
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Patients’ view on gene therapy development for lysosomal storage disorders: a qualitative study
Eskes, E. C. B., Beishuizen, C. R. L., Corazolla, E. M., van Middelaar, T., Brands, M. M. M. G., Dekker, H., van de Mheen, E., Langeveld, M., Hollak, C. E. M. & Sjouke, B., 1 Dec 2022, In: Orphanet journal of rare diseases. 17, 1, 383.Research output: Contribution to journal › Article* › Academic › peer-review
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