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What We Do

Rare Diseases

Audiences

  • Endocrinologists

  • Geneticists

  • Hematologists

  • Individual rare disease centers (i.e. hemophilia)

  • Individual rare disease chapters and support groups

  • Pathologists

  • Infectious disease specialists

  • Patients and caregivers

  • Nephrologists

  • Rheumatologists

Topics

  • Bone marrow transplants

  • Coagulation disorders (Hemophilia)

  • Alpha- 1

  • Fabry

  • Gaucher

  • Hypophosphatasia

  • Pompe

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Research Expertise