Real-life impacts of olipudase alfa: experiences of adults receiving enzyme replacement therapy for acid sphingomyelinase deficiency-results from an international survey study.
- DOI
- 10.1186/s13023-025-03997-6
- Published
- 2025 Sep 30
- Container
- Orphanet journal of rare diseases
- Publisher
- Not recorded
- Open access
- yes
Credibility signals
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Cite this work
BibTeX
@article{allodium:10.1186/s13023-025-03997-6,
title = {Real-life impacts of olipudase alfa: experiences of adults receiving enzyme replacement therapy for acid sphingomyelinase deficiency-results from an international survey study.},
author = {Morsy AS and Mbua S and Mathieson T and Hopkin J and Bolton S},
year = {2025},
journal = {Orphanet journal of rare diseases},
doi = {10.1186/s13023-025-03997-6},
url = {https://doi.org/10.1186/s13023-025-03997-6}
}RIS
TY - JOUR TI - Real-life impacts of olipudase alfa: experiences of adults receiving enzyme replacement therapy for acid sphingomyelinase deficiency-results from an international survey study. AU - Morsy AS AU - Mbua S AU - Mathieson T AU - Hopkin J AU - Bolton S PY - 2025 JO - Orphanet journal of rare diseases DO - 10.1186/s13023-025-03997-6 UR - https://doi.org/10.1186/s13023-025-03997-6 ER -
APA
AS, M., S, M., T, M., J, H., & S, B. (2025). Real-life impacts of olipudase alfa: experiences of adults receiving enzyme replacement therapy for acid sphingomyelinase deficiency-results from an international survey study.. Orphanet journal of rare diseases. https://doi.org/10.1186/s13023-025-03997-6
Source records
- pubmed · retrieved 2026-09-27T08:17:38.246Z
- europe-pmc · retrieved 2026-09-27T08:17:38.246Z
- doaj · retrieved 2026-09-27T08:17:38.256Z