The AAMDSIF Leadership in Science Award was presented to Amy DeZern, MD, MHS, at our 8th International Bone Marrow Failure Bone Marrow Failure: A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be… Disease Scientific Symposium. Dr. DeZern is Professor of Oncology and Director of the Bone Marrow Failure and MDS Program at the Johns Hopkins Sidney Kimmel Comprehensive Cancer Center in Baltimore, Maryland. Her research has focused on designing and conducting clinical trials clinical trials: Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect or treat disease. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study. Understanding Clinical Trials Clinical… to investigate novel therapies and approaches in the treatment of aplastic anemia aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… and MDS. Dr. DeZern’s work is significantly changing the standard of care for aplastic anemia, with specific expertise in the treatment of relapsed and refractory refractory: Not responsive to treatment or cure. For example, refractory anemia is a low red blood cell count that doesn't respond to standard treatments. severe aplastic anemia and the use of alternative donor bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. transplantation. She has been a Principal Investigator for numerous MDS clinical trials and collaborative research studies to develop new therapies and translate them to clinical practice. Dr. DeZern has also demonstrated the role of specialized testing to distinguish cellular marrow failure disorders as well as genetic evaluations for patients with both inherited and acquired bone marrow failure. She has been a member of the AAMDSIF Medical Advisory Board since 2013.