Jeffrey W Olin, DO, Professor of Medicine at the Icahn School of Medicine at Mount Sinai, discusses diagnosing and treating fibromuscular dysplasia (FMD), a rare condition characterized by abnormal development or growth of cells in the walls of blood vessels (arteries) that can cause the vessels to narrow.
Ido Weinberg, MD, Medical Director, VASCORE; Assistant Professor of Medicine, Harvard Medical School, Co-Medical Director, Anticoagulation Management Services, Massachusetts General Hospital, discusses the importance of raising awareness of fibromuscular dysplasia (FMD) and having patients visit centers of excellence for their treatments.
Anne R. Pariser, MD, Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences at the National Institutes of Health discusses gene therapy and how the NIH is looking at “platform approaches” for treatments for rare diseases.
Malissa J. Wood, MD, is co-director of the Corrigan Women’s Heart Health Program at Massachusetts General Hospital and associate professor of medicine at Harvard Medical School. In this video, Dr. Wood discusses the relationship between Fibromuscular Dysplasia (FMD) and Spontaneous Coronary Artery Dissection (SCAD).
The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to CAD-1883, an investigational treatment for spinocerebellar ataxia (SCA), a genetic disorder characterized by progressive loss of coordination, slurred speech, difficulty controlling eye movements and cognitive dysfunction.
The US Food and Drug Administration granted orphan drug designation for AT-007, a central nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI) in the late stages of preclinical development for treating children with galactosemia.