Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best–worst scaling experiment in caregivers and adult patients

PURPOSE: Several gene therapy trials for Duchenne muscular dystrophy initiated in 2018. Trial decision making is complicated by non-curative, time-limited benefits; the progressive, fatal course; and high unmet needs. Here, caregivers and patients prioritize factors influencing decision making regar...

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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: Paquin, Ryan S., Fischer, Ryan, Mansfield, Carol, Mange, Brennan, Beaverson, Katherine, Ganot, Annie, Martin, Amy Strong, Morris, Carl, Rensch, Colin, Ricotti, Valeria, Russo, Leo J., Sadosky, Alesia, Smith, Edward C., Peay, Holly L.
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2019
Assuntos:
Research
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6509771/
https://ncbi.nlm.nih.gov/pubmed/31072340
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-019-1069-6
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