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The Dystroglycanopathies : 2013 Patient and Family Conference PowerPoint Presentation
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The Dystroglycanopathies : 2013 Patient and Family Conference

The Dystroglycanopathies : 2013 Patient and Family Conference

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The Dystroglycanopathies : 2013 Patient and Family Conference

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  1. The Dystroglycanopathies: 2013 Patient and Family Conference Carrie Stephan

  2. The Early Days • A Clinical Evaluation of the FKRP Muscular Dystrophies • ‘FKRP Study’ • describe signs & symptoms • determine reasons for differences in disease severity

  3. 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

  4. Total enrollment per year

  5. Conference Attendance