Rare and Genetic Disease Network
Featured
Rare Disease Film Festival Coming to NYC
This May 18th, Chelsea in New York will host DISORDER, the rare disease film festival.
Phase II Study Testing Losmapimod for Facioscapulohumeral Muscular Dystrophy
Diego Cadavid, MD of Fulcrum Therapeutics explains the ongoing phase II clinical trial for facioscapulohumeral muscular dystrophy.
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 is a rare, congenital primary immunodeficiency disorder associated with severe neutropenia that affects all ages.
Rare Disease Patients – Their Time Has Come
Priya Kishnani, MD, of Duke University School of Medicine speaks to the rare disease patient community and says ‘their time has come’.
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
Dr. John Mitchell explains the pathophysiology, symptoms, and treatment options for Farber disease.
WORLDSymposium: Then and Now
John Crowley, CEO and Chairman at Amicus Therapeutics talks about the growth of WORLDSymposium over the past 16 years.
Latest Results from the Morquio A Registry Study (MARS)
Barbara Burton, MD of Lurie Children’s Hospital of Chicago discusses the latest results from the Morquio A Registry Study (MARS).
Gene Therapy Clinical Trial Showing Promising Results for Pompe Disease
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.
p38 inhibition to Treat FSHD: Turning Off DUX4
Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common forms of muscular dystrophy.
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
Tenosynovial Giant Cell Tumor (TGCT)
Clinical Trials and Rare Diseases
Accelerating the Road to Treatment: The Global Commission to End the Diagnostic Odyssey for Children
Mucopolysaccharidosis (MPS) Resource Center
Overview of the Mucopolysaccharidoses
Rare in Common Podcast
American Society of Hematology Conference Coverage
Considering Rare Diseases for Challenging Cases
Duchenne Muscular Dystrophy
Rare Disease Diagnosis: Huntington’s Disease
Tenosynovial Giant Cell Tumor (TGCT)
Clinical Trials and Rare Diseases
Accelerating the Road to Treatment: The Global Commission to End the Diagnostic Odyssey for Children
Mucopolysaccharidosis (MPS) Resource Center
Overview of the Mucopolysaccharidoses
Rare in Common Podcast
American Society of Hematology Conference Coverage
Considering Rare Diseases for Challenging Cases
Duchenne Muscular Dystrophy
