by Peter Ciszewski | Feb 27, 2020
This May 18th, Chelsea in New York will host DISORDER, the rare disease film festival. This will be the third DISORDER festival that provides an opportunity for filmmakers to share their rare disease stories with a live audience of patients, advocates, researchers,...
by Peter Ciszewski | Feb 26, 2020
Diego Cadavid, MD of Fulcrum Therapeutics explains the ongoing phase II clinical trial for facioscapulohumeral muscular dystrophy. Facioscapulohumeral muscular dystrophy is a disabling disease characterized by progressive skeletal muscle loss. The rare disease...
by Peter Ciszewski | Feb 24, 2020
Gallia Levy, MD, PhD, Associate Group Medical Director at Genentech, explains treatment options for hemophilia A. Hemophilia A is a genetic disorder caused by missing or defective clotting protein, factor VIII. Symptoms are dependent on the level factor VIII...
by Peter Ciszewski | Feb 21, 2020
Priya Kishnani, MD, Duke University School of Medicine, speaks to the rare disease patient community and says ‘their time has come’. Dr. Kishnani was speaking largely to the patient advocacy groups associated with lysosomal storage diseases, like Pompe disease,...
by Peter Ciszewski | Feb 18, 2020
Farzana Sayani, MD, Assistant Professor of Medicine at the Hospital of the University of Pennsylvania explains the pathophysiology of sickle cell disease. Sickle cell disease is an inherited blood disorder in which the red blood cells are abnormally sickle...
