by Peter Ciszewski | Nov 18, 2022
Mark Forman, MD, PhD, Chief Medical Officer at Passage Bio, gives an overview of frontotemporal dementia (FTD) with granulin (GRN) mutations. As Dr. Forman explains, FTD is a disorder that affects the frontal and temporal lobes of the brain, areas that control...
by Peter Ciszewski | Nov 17, 2022
Eva Morava-Kozicz, MD, PhD of the Mayo Clinic in Rochester, MN profiles an overview of congenital disorders of glycosylation (CDG). CDG are rare genetic disorders that impact glycosylation, a natural process in which sugar molecules are attached to proteins...
by Peter Ciszewski | Nov 16, 2022
Angela Scheuerle, MD, Medical Geneticist at the UT Southwestern Medical Center, discusses National Organization for Rare Disorders’ (NORD) Rare Disease Centers of Excellence of which UT Southwestern is one. There are currently 31 NORD Rare Disease Centers of...
by Peter Ciszewski | Nov 14, 2022
Annette von Drygalski, MD, PharmD, Associate Clinical Professor of Medicine in the Division of Hematology/Oncology, and Director of the Hemophilia and Thrombosis Treatment Center, Department of Medicine, at the University of California, San Diego, discusses the...
by Peter Ciszewski | Nov 10, 2022
Donald Zoz, MD, Director and Senior Clinical Program Leader for Pulmonary Fibrosis at Boehringer Ingelheim explains the mechanism of action of BI 1015550 to treat idiopathic pulmonary fibrosis (IPF). IPF is a chronic, progressive, fibrosing lung disease with...