Fabry Disease.

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Presentation transcript:

Fabry Disease

Fabry Disease Pattern of Inheritance

Clinical Manifestations of Fabry Disease

Recommended Schedule of Assessments for Pediatric Patients From the Fabry Registry

Recommended Schedule of Assessments for Adult Patients From the Fabry Registry

Genetic Mutations Linked to Fabry Disease

Enzyme Replacement Therapy (ERT)

Instituting ERT in Patients With Fabry Disease

ERT With Agalsidase Beta Ten-Year Outcome

Initiating ERT in Pediatric Patients With Fabry Disease

ATTRACT Phase 3 Study of Migalastat vs ERT

Abbreviations