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Development of Barth Syndrome Questionnaire for Research

Jonathan Stokes, MBA, Director Patient Centered Outcomes Adelphi Values This webinar provided an update to families on the development of questionnaires to be used in future Stealth BioTherapeutics clinical trials to evaluate the effectiveness of a new Barth Syndrome treatment. In July, members of the Adelphi Values team attended the BSF conference held in Clearwater, FL to speak with individuals to better understand the Barth Syndrome experience. We want to sincerely thank everyone for their time and willingness to participate in this important research. Based on findings from those discussions, two symptom-based questionnaires were created. As part of this webinar, we would like to share the findings of this research. ADELPHI VALUES, 2017. Adelphi Values holds copyright to the concepts, statements and formats described in this presentation. The unauthorized use of any portion of this presentation is prohibited.