
What We Do
Rare Diseases
Audiences
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Endocrinologists
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Geneticists
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Hematologists
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Individual rare disease centers (i.e. hemophilia)
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Individual rare disease chapters and support groups
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Pathologists
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Infectious disease specialists
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Patients and caregivers
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Nephrologists
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Rheumatologists
Topics
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Bone marrow transplants
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Coagulation disorders (Hemophilia)
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Alpha- 1
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Fabry
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Gaucher
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Hypophosphatasia
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Pompe
