Patients’ and caregivers’ maximum acceptable risk of death for non‐curative gene therapy to treat Duchenne muscular dystrophy

BACKGROUND: Gene therapy offers an etiologically targeted treatment for genetic disorders. Little is known about the acceptance of mortality risk among patients with progressive, fatal conditions. We assessed patients’ and caregivers’ maximum acceptable risk (MAR) of mortality for gene therapy when...

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Detalles Bibliográficos
Autores principales: Peay, Holly L., Fischer, Ryan, Mange, Brennan, Paquin, Ryan S., Smith, Edward C., Sadosky, Alesia, Russo, Leo, Ricotti, Valeria, Rensch, Colin, Morris, Carl, Martin, Amy Strong, Ganot, Annie, Beaverson, Katherine, Mansfield, Carol
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2021
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8172191/
https://www.ncbi.nlm.nih.gov/pubmed/33755338
http://dx.doi.org/10.1002/mgg3.1664

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