Rare and Genetic Disease Network
This May 18th, Chelsea in New York will host DISORDER, the rare disease film festival.
Diego Cadavid, MD of Fulcrum Therapeutics explains the ongoing phase II clinical trial for facioscapulohumeral muscular dystrophy.
Gallia Levy, MD, PhD, Associate Group Medical Director at Genentech, explains treatment options for hemophilia A.
WHIM syndrome is a rare, congenital primary immunodeficiency disorder associated with severe neutropenia that affects all ages.
Priya Kishnani, MD, of Duke University School of Medicine speaks to the rare disease patient community and says ‘their time has come’.
Farzana Sayani, MD, Assistant Professor of Medicine at the Hospital of the University of Pennsylvania explains the pathophysiology of sickle cell disease.
Dr. John Mitchell explains the pathophysiology, symptoms, and treatment options for Farber disease.
John Crowley, CEO and Chairman at Amicus Therapeutics talks about the growth of WORLDSymposium over the past 16 years.
Barbara Burton, MD of Lurie Children’s Hospital of Chicago discusses the latest results from the Morquio A Registry Study (MARS).
Dwight Koebel, MD, PhD, of Duke University School of Medicine provides an update on the phase 1 study of gene therapy to treat late-onset Pompe disease.
Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common forms of muscular dystrophy.
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.