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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2019
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6509771/
https://www.ncbi.nlm.nih.gov/pubmed/31072340
http://dx.doi.org/10.1186/s13023-019-1069-6

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