Rare and Genetic Disease Network

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Hemlibra to Treat Hemophilia A

Hemlibra to Treat Hemophilia A

Gallia Levy, MD, PhD, Associate Group Medical Director at Genentech, explains treatment options for hemophilia A.

WHIM Syndrome

WHIM Syndrome

WHIM syndrome is a rare, congenital primary immunodeficiency disorder associated with severe neutropenia that affects all ages.

Sickle Cell Disease Explained

Sickle Cell Disease Explained

Farzana Sayani, MD, Assistant Professor of Medicine at the Hospital of the University of Pennsylvania explains the pathophysiology of sickle cell disease.

Farber Syndrome Explained

Farber Syndrome Explained

Dr. John Mitchell explains the pathophysiology, symptoms, and treatment options for Farber disease.

WORLDSymposium: Then and Now

WORLDSymposium: Then and Now

John Crowley, CEO and Chairman at Amicus Therapeutics talks about the growth of WORLDSymposium over the past 16 years.

Castleman Disease Explained

Castleman Disease Explained

Ruth-Anne Langan Pai, PhD Candidate and the collaborative research project lead for the Castleman Disease Collaborative Network (CDCN) provides an overview of Castleman disease.

Special Reports

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