Sandy's Long, Hard, Road
I was diagnosed with Severe 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
treatment
Alrizomadlin (APG-115)
Alrizomadlin is an orally administered, selective, small-molecule inhibitor of the MDM2 protein. Alrizomadlin has strong binding affinity to MDM2 and is designed to activate tumor suppression activity of p53 by blocking the MDM2-p53 protein-protein interaction.
Lisaftoclax (APG-2575)
APG-2575 is a novel, orally administered small-molecule Bcl-2‒selective inhibitor.
Gerard Socie, MD
Institution
Hospital St. Louis
Physician Status
accepting new patients
Primary Disease Area of Focus
Aplastic Anemia
Graft Versus Host Disease (GVHD)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
About
Gerard Socié, MD, PhD, is the head of the hematology department at Hospital Saint-Louis, APHP. Gerard Socié has a research appointment at the Institut Pasteur -APHP with Dr. Lars Rogge. His main fields of expertise are transplantation (GvHD), aplastic anemia, PNH and Fanconi Anemia. Dr. Socié discusses the diagnosis, treatment and prognosis of PNH in this pre-recorded session.
Sioban Keel, MD
Institution
Fred Hutchinson Cancer Research Center
Physician Status
accepting new patients
Primary Disease Area of Focus
Aplastic Anemia
Paroxysmal Nocturnal Hemoglobinuria (PNH)
About
Sioban Keel, M.D., is a board certified hematologist at SCCA and a UW associate professor of Hematology and Medicine. Dr. Keel believes that excellent medical care requires a combination of up-to-date knowledge and compassionate, individualized patient care. She recognizes that excellent care requires physicians who communicate effectively with and educate each patient so that they understand their condition and can be a partner in determining the best treatment plan. Dr. Keel earned her M.D. at the University of Minnesota. Her clinical and research interests include benign red blood cell
Alexion Announces FDA Approval of ULTOMIRIS® (ravulizumab-cwvz) for Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH)
Original Publication Date
Article Source
External Web Content
– With this approval, ULTOMIRIS is the first and only medicine approved in the U.S. to treat children and adolescents with PNH – – Approval based on interim results from Phase 3 study showing ULTOMIRIS demonstrated complete terminal complement inhibition through 26 weeks – BOSTON…
Living with GVHD: A Transplant Patient Panel Discussion
Topic(s)
Acute Myeloid Leukemia (AML)
Aplastic Anemia
Bone Marrow Transplant
Living Well with Bone Marrow Failure
Managing Your Treatment
Myelodysplastic Syndromes (MDS)
Related Bone Marrow Failure Diseases and Malignancies
Presenter(s)
Leigh Clark
Patient panel discussions are a chance to meet some of the inspiring patients, caregivers and parents who are dealing with a bone marrow failure disease. Panel discussions are a bit different from our regular educational webinars so please plan to participate via the Q & A window…
Clinical effects of administering leukemia-specific donor T cells to patients with AML/MDS after allogeneic transplant
Original Publication Date
Article Source
External Web Content
Key Points Donor-derived T cells with native specificity for multiple myeloid leukemia antigens can be expanded ex vivo. Infusion of mLSTs after HCT is well tolerated and produces antileukemia effects. Abstract Relapse after allogeneic hematopoietic stem cell transplantation (HCT…
Venetoclax
Venetoclax Venetoclax: Venetoclax is used to treat chronic lymphocytic leukemia or small lymphocytic leukemia in adults.
Global PNH Patient Registry
The Global PNH Patient Registry is a collaborative effort between the