Disease characteristics, effectiveness, and safety of vestronidase alfa for the treatment of patients with mucopolysaccharidosis VII in a novel, longitudinal, multicenter disease monitoring program

Roberto Giugliani, Antonio Gonzalez-Meneses, Maurizio Scarpa, Barbara Burton, Raymond Wang, Esmeralda Martins, Esmeralda Oussoren, Julia B. Hennermann, Brigitte Chabrol, Christina L. Grant, Angela Sun, Consuelo Durand, Joel Hetzer, Betsy Malkus, Deborah Marsden, J. Lawrence Merritt II

Open source

DOI: 10.1186/s13023-024-03176-z
Published: 2024-05-07
Container: Orphanet Journal of Rare Diseases
Publisher: Springer Science and Business Media LLC
Open access: unknown

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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 = {Roberto Giugliani and Antonio Gonzalez-Meneses and Maurizio Scarpa and Barbara Burton and Raymond Wang and Esmeralda Martins and Esmeralda Oussoren and Julia B. Hennermann and Brigitte Chabrol and Christina L. Grant and Angela Sun and Consuelo Durand and Joel Hetzer and Betsy Malkus and Deborah Marsden and J. Lawrence Merritt II},
  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  - Roberto Giugliani
AU  - Antonio Gonzalez-Meneses
AU  - Maurizio Scarpa
AU  - Barbara Burton
AU  - Raymond Wang
AU  - Esmeralda Martins
AU  - Esmeralda Oussoren
AU  - Julia B. Hennermann
AU  - Brigitte Chabrol
AU  - Christina L. Grant
AU  - Angela Sun
AU  - Consuelo Durand
AU  - Joel Hetzer
AU  - Betsy Malkus
AU  - Deborah Marsden
AU  - J. Lawrence Merritt II
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

Giugliani, R., Gonzalez-Meneses, A., Scarpa, M., Burton, B., Wang, R., Martins, E., Oussoren, E., Hennermann, J. B., Chabrol, B., Grant, C. L., Sun, A., Durand, C., Hetzer, J., Malkus, B., Marsden, D., & II, J. L. M. (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

crossref · retrieved 2026-11-10T18:13:18.206Z