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Understanding and Treating Dystroglycanopathies: A Comprehensive Approach

Join us at the 2013 Patient and Family Conference to explore the FKRP Muscular Dystrophies, clinical trial readiness, and advancements in disease understanding. Experts will discuss signs and symptoms, disease severity differences, longitudinal design, and natural history. Be part of refining tools for future treatments.

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Understanding and Treating Dystroglycanopathies: A Comprehensive Approach

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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

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