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The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor

key information

study id #: NCT01182324

condition: Duchenne Muscular Dystrophy

status: completed


The purpose of this study is to describe the experiences of parents, clinician researchers, and industry professionals who were involved in phase II clinical trials of Ataluren for Duchenne muscular dystrophy. We are especially interested in learning about motivations for being involved in the clinical trial, expectations of the trial, the experience of the trial, and relationships between the parents of children involved in the trial, the clinician researchers, and PTC Therapeutics. In addition, we would like to learn more about whether and how families and advocacy organizations experiences in following the progress of the drug, encouraging the clinical trial, and supporting the phase II trials may have affected participants thoughts and feelings about the study.

mechanism of action: Ribosomal alterator to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT01182324

last updated: November 22, 2018