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Expectations and experiences of investigators and parents involved in a clinical trial for Duchenne/Becker muscular dystrophy

BACKGROUND: The social context of rare disease research is changing, with increased community engagement around drug development and clinical trials. This engagement may benefit patients and families, but may also lead to heightened trial expectations and therapeutic misconception. Clinical investig...

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Autori principali: Peay, Holly L, Tibben, Aad, Fisher, Tyler, Brenna, Ethan, Biesecker, Barbara B
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2013
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3935382/
https://ncbi.nlm.nih.gov/pubmed/24311736
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1177/1740774513512726
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