Different diseases, different needs: Patient preferences for gene therapy in lysosomal storage disorders, a probabilistic threshold technique survey
People affected by a lysosomal storage disease associated with a poorer prognosis and less effective current treatment options trended towards more risk tolerance when choosing between gene therapy and the current standard of care. This study shows the importance of involvement of patient preferences before and during the development process of new treatment modalities such as gene therapy for rare diseases, to ensure that innovative therapies align with the wishes and needs of people affected by these diseases.
Authors
- Eline C. B. Eskes (ORCID: https://orcid.org/0000-0003-3662-0212)
- Barbara Sjouke (ORCID: https://orcid.org/0000-0001-8162-9684)
- Carla E. M. Hollak (ORCID: https://orcid.org/0000-0003-0464-1078)
- Hanka Dekker (ORCID: https://orcid.org/0009-0009-4284-9129)
- Mirjam Langeveld (ORCID: https://orcid.org/0000-0002-9934-6831)
- Marion M. Brands (ORCID: https://orcid.org/0000-0002-2025-5997)
- Eleonore M. Corazolla (ORCID: https://orcid.org/0000-0003-2787-2549)
- Jorien Veldwijk (ORCID: https://orcid.org/0000-0003-4822-5068)
- Erica van de Mheen
Institutions
- Netherlands Institute for Neuroscience (NL)
- Radboud University Nijmegen (NL)
- Radboud University Medical Center (NL)
- Emma Kinderziekenhuis (NL)
- Radboud Institute for Molecular Life Sciences (NL)
- Association of Dutch Burn Centres (NL)
- Amsterdam University Medical Centers (NL)
- University of Amsterdam (NL)
- Erasmus University Rotterdam (NL)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2024-10-03
- DOI
- https://doi.org/10.1186/s13023-024-03371-y
- Citations
- 4
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 1.31
Funders
- Amsterdam University Medical Centers