Disease characteristics, effectiveness, and safety of vestronidase alfa for the treatment of patients with mucopolysaccharidosis VII in a novel, longitudinal, multicenter disease monitoring program.
- DOI
- 10.1186/s13023-024-03176-z
- Published
- 2024 May 7
- Container
- Orphanet journal of rare diseases
- Publisher
- Not recorded
- Open access
- yes
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Cite this work
BibTeX
@article{allodium:10.1186/s13023-024-03176-z,
title = {Disease characteristics, effectiveness, and safety of vestronidase alfa for the treatment of patients with mucopolysaccharidosis VII in a novel, longitudinal, multicenter disease monitoring program.},
author = {Giugliani R and Gonzalez-Meneses A and Scarpa M and Burton B and Wang R and Martins E and Oussoren E and Hennermann JB and Chabrol B and Grant CL and Sun A and Durand C and Hetzer J and Malkus B and Marsden D and Merritt Ii JL},
year = {2024},
journal = {Orphanet journal of rare diseases},
doi = {10.1186/s13023-024-03176-z},
url = {https://doi.org/10.1186/s13023-024-03176-z}
}RIS
TY - JOUR TI - Disease characteristics, effectiveness, and safety of vestronidase alfa for the treatment of patients with mucopolysaccharidosis VII in a novel, longitudinal, multicenter disease monitoring program. AU - Giugliani R AU - Gonzalez-Meneses A AU - Scarpa M AU - Burton B AU - Wang R AU - Martins E AU - Oussoren E AU - Hennermann JB AU - Chabrol B AU - Grant CL AU - Sun A AU - Durand C AU - Hetzer J AU - Malkus B AU - Marsden D AU - Merritt Ii JL PY - 2024 JO - Orphanet journal of rare diseases DO - 10.1186/s13023-024-03176-z UR - https://doi.org/10.1186/s13023-024-03176-z ER -
APA
R, G., A, G., M, S., B, B., R, W., E, M., E, O., JB, H., B, C., CL, G., A, S., C, D., J, H., B, M., D, M., & JL, M. I. (2024). Disease characteristics, effectiveness, and safety of vestronidase alfa for the treatment of patients with mucopolysaccharidosis VII in a novel, longitudinal, multicenter disease monitoring program.. Orphanet journal of rare diseases. https://doi.org/10.1186/s13023-024-03176-z
Source records
- pubmed · retrieved 2026-09-26T15:21:30.046Z