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