Re-evaluating acceptable risk of death from gene therapy: A threshold study among individuals with Duchenne muscular dystrophy and their caregivers in the US and UK

Holly Peay, Ryan Fischer, Megan McNiff, Emma Heslop, Anna Pierce, Brian Denger, Eric Camino, Alexandra Johnson, Heidi Cope, Christine Hill, Katherine L. Beaverson, Annie Ganot, Dawn Phillips, Ione O.C. Woollacott, Alison Bateman-House, Kevin M. Flanigan, Nathalie Goemans, Laurent Servais, Michela Guglieri, Carol Mansfield

Open source

DOI: 10.1016/j.ejmg.2025.105050
Published: 2025-12
Container: European Journal of Medical Genetics
Publisher: Elsevier BV
Open access: unknown

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Cite this work

BibTeX

@article{allodium:10.1016/j.ejmg.2025.105050,
  title = {Re-evaluating acceptable risk of death from gene therapy: A threshold study among individuals with Duchenne muscular dystrophy and their caregivers in the US and UK},
  author = {Holly Peay and Ryan Fischer and Megan McNiff and Emma Heslop and Anna Pierce and Brian Denger and Eric Camino and Alexandra Johnson and Heidi Cope and Christine Hill and Katherine L. Beaverson and Annie Ganot and Dawn Phillips and Ione O.C. Woollacott and Alison Bateman-House and Kevin M. Flanigan and Nathalie Goemans and Laurent Servais and Michela Guglieri and Carol Mansfield},
  year = {2025},
  journal = {European Journal of Medical Genetics},
  doi = {10.1016/j.ejmg.2025.105050},
  url = {https://doi.org/10.1016/j.ejmg.2025.105050}
}

RIS

TY  - JOUR
TI  - Re-evaluating acceptable risk of death from gene therapy: A threshold study among individuals with Duchenne muscular dystrophy and their caregivers in the US and UK
AU  - Holly Peay
AU  - Ryan Fischer
AU  - Megan McNiff
AU  - Emma Heslop
AU  - Anna Pierce
AU  - Brian Denger
AU  - Eric Camino
AU  - Alexandra Johnson
AU  - Heidi Cope
AU  - Christine Hill
AU  - Katherine L. Beaverson
AU  - Annie Ganot
AU  - Dawn Phillips
AU  - Ione O.C. Woollacott
AU  - Alison Bateman-House
AU  - Kevin M. Flanigan
AU  - Nathalie Goemans
AU  - Laurent Servais
AU  - Michela Guglieri
AU  - Carol Mansfield
PY  - 2025
JO  - European Journal of Medical Genetics
DO  - 10.1016/j.ejmg.2025.105050
UR  - https://doi.org/10.1016/j.ejmg.2025.105050
ER  -

APA

Peay, H., Fischer, R., McNiff, M., Heslop, E., Pierce, A., Denger, B., Camino, E., Johnson, A., Cope, H., Hill, C., Beaverson, K. L., Ganot, A., Phillips, D., Woollacott, I. O., Bateman-House, A., Flanigan, K. M., Goemans, N., Servais, L., Guglieri, M., & Mansfield, C. (2025). Re-evaluating acceptable risk of death from gene therapy: A threshold study among individuals with Duchenne muscular dystrophy and their caregivers in the US and UK. European Journal of Medical Genetics. https://doi.org/10.1016/j.ejmg.2025.105050

Source records

crossref · retrieved 2026-09-27T12:47:32.057Z