by Peter Ciszewski | Jun 10, 2020
Bettina Cockcroft, MD, Chief Medical Officer at Sangamo Therapeutics, recently talked to CheckRare about the gene therapy that the company has in development to treat people with Fabry disease. Fabry disease is a rare lysosomal storage disorder involving mutations of...
by Peter Ciszewski | Jun 9, 2020
Courtney DiNardo, MD, Associate Professor at the University of Texas MD Anderson Cancer Center explains the results of a recent study assessing the safety and efficacy of venetoclax combination therapies in elderly patients with acute myeloid leukemia (AML)....
by Peter Ciszewski | Jun 8, 2020
The Global Commission to End the Diagnostic Odyssey for Children with a Rare Disease is a multidisciplinary collaboration of experts from around the world co-chaired by Takeda, Microsoft and EURORDIS-Rare Diseases Europe, who have brought together their...
by Peter Ciszewski | Jun 8, 2020
Kathleen Hawker, MD, Group Medical Director at Genentech, describes the pathophysiology and common symptoms of neuromyelitis optica spectrum disease (NMOSD), and how they differ from multiple sclerosis. NMOSD is a rare autoimmune disease of the central nervous...
by Peter Ciszewski | Jun 5, 2020
David Dale, MD at the University of Washington Medical Center discusses quality of life issues with patients who suffer from rare genetic disorders like WHIM syndrome. WHIM syndrome is a rare, congenital primary immune disorder that affects all ages. However,...
