Transcript
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The Dystroglycanopathies: 2013 Patient and Family Conference
Carrie Stephan
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The Early Days
•A Clinical Evaluation of the FKRP Muscular Dystrophies
• ‘FKRP Study’
• describe signs & symptoms
• determine reasons for differences in disease severity
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Clinical Trial Readiness for the Dystroglycanopathies
• ‘Wellstone Study’
• Longitudinal design
• Conducted in preparation for a clinical trial• Identify patients• Describe signs & symptoms• Define natural history and refine tools
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Total enrollment per year
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Conference Attendance