Fighting for a Transplant : Stefania conquers Aplastic Anemia and PNH ~ Stefania demuestra su VERDADERA fuerza
Hi! My name is Stefania, I am 31 years old, and I am from Ecuador.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
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
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…
Pediatric PNH - Diagnosis and Treatment
Topic(s)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Presenter(s)
Satheesh Chonat, MD
In this webinar, Dr. Satheesh Chonat will discuss what is PNH, how PNH is diagnosed and what treatment options are available to pediatric patients. After the presentation, Dr. Chonat will answer questions from participants. Advance questions can be submitted to help@aamds.org.
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…
Clinical Trials in Depth
Clinical trials Clinical trials: Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect or treat disease.
Allyson Pishko, MD
Institution
University of Pennsylvania
Physician Status
available for consultation
Primary Disease Area of Focus
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Aspaveli® (pegcetacoplan) approved in EU as orphan drug for treatment of PNH
Original Publication Date
Article Source
External Web Content
Published: Dec 15, 2021 STOCKHOLM, Dec. 15, 2021 /PRNewswire/ -- The first targeted C3 therapy approved in the EU Approval based on results from head-to-head PEGASUS phase 3 study where Aspaveli demonstrated superiority to eculizumab in improving haemoglobin levels1 Aspaveli will…