Aplastic Anemia

Science Simplified: What is a Natural History Study?

Original Publication Date
Article Source
External Web Content
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science accessible and empower rare disease community members with scientific knowledge. Dr…

Emma Groarke, MD, FRCPath

Institution
National Lung, Heart and Blood Institute (NLHBI)
Physician Status
available for consultation
Primary Disease Area of Focus
Aplastic Anemia
Myelodysplastic Syndromes (MDS)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Pure Red Cell Aplasia (PRCA)
About
Dr. Emma M. Groarke is a hematologist in Bethesda, Maryland at the National Lung, Heart and Blood Insititute. She received her medical degree from University College of Dublin. Dr. Groarke research involves Clinical trials in acquired and inherited bone marrow failure.

Genetic Testing for Bone Marrow Failure Diseases

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Topic(s)
Living Well with Bone Marrow Failure
Managing Your Treatment
Presenter(s)
Jadee Neff, M.D., Ph.D., FCAP
In this webinar, Dr. Jadee Neff discusses how genetic testing used in the diagnostic process, how it can help identify possible causes of disease that inform decisions regarding appropriate therapies, identify potential donors for bone marrow transplant, and potential risk for…

Satheesh Chonat, MD

Institution
Children’s Healthcare of Atlanta
Physician Status
available for consultation
Primary Disease Area of Focus
Aplastic Anemia
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Pediatric
About
Satheesh Chonat, MD, is a Pediatric Hematologist and Oncologist at Children’s Healthcare of Atlanta and board-certified in pediatrics and pediatric hematology/oncology. He specializes in the care of children and young adults with nonmalignant rare blood disorders by using a variety of clinical and research-based tools. Dr. Chonat is the site investigator for various clinical and translational research projects, focused on rare red blood disorders, such as pyruvate kinase disease, thrombotic thrombocytopenic purpura and paroxysmal nocturnal hemoglobinuria. The main focus of his laboratory

Clinical Trials and Children

Until recently, children were rarely included in studies of medical treatments. As a result, much is still unknown about how children respond to drugs, some biologics (such as gene therapy) and medical devices. According to the U.S. Food and Drug Administration (FDA), only 20-30% of approved drugs have actually been labeled for use in children.

mRNA COVID-19 Vaccination Sparks Strong Antibody Response in Patients With AML and MDS

Original Publication Date
Article Source
External Web Content
The mRNA COVID-19 Vaccines Are Safe and Effective for Most Patients with Cancer For most patients with cancer, the mRNA COVID-19 vaccines are safe and effective. This article explains the results of the first study that evaluated the safety and effectiveness of the mRNA vaccines…
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