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

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

Abstract 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 thera...

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Bibliographic Details
Main Authors: Holly L. Peay, Ryan Fischer, Brennan Mange, Ryan S. Paquin, Edward C. Smith, Alesia Sadosky, Leo Russo, Valeria Ricotti, Colin Rensch, Carl Morris, Amy Strong Martin, Annie Ganot, Katherine Beaverson, Carol Mansfield
Format: Article
Language:English
Published: Wiley 2021-05-01
Series:Molecular Genetics & Genomic Medicine
Subjects:
Duchenne muscular dystrophy
gene therapy
patient‐reported outcomes
risk
Online Access:https://doi.org/10.1002/mgg3.1664

Internet

https://doi.org/10.1002/mgg3.1664

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