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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Detaylı Bibliyografya
Yayımlandı:Mol Genet Genomic Med
Asıl Yazarlar: 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
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: John Wiley and Sons Inc. 2021
Konular:
Original Articles
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC8172191/
https://ncbi.nlm.nih.gov/pubmed/33755338
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/mgg3.1664
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