Paroxysmal Nocturnal Hemoglobinuria (PNH)
David Young, MD, PhD
Dr. Young is a board-certified Pediatrician and Pediatric Hematologist and Oncologist who studies the genetics, molecular biology, and clinical management and treatment of bone marrow failure syndromes, myeloid malignancies, and other genetic disorders. He oversees clinical trials in aplastic anemia and Diamond-Blackfan anemia while also working with non-human primate models of disease and preclinical development. He also serves as the Medical Director for the RUNX1 Natural History Program of the National Human Genome Research Institute. Dr. Young received a B.Sc. from the University of Notre
Imputing causality and clonal dynamics from single-cell transcriptomics in paroxysmal nocturnal hemoglobinuria
Abstract Paroxysmal nocturnal hemoglobinuria (PNH) originates from hematopoietic stem cells (HSCs) harboring somatic mutations in the phosphatidylinositol glycan class A (PIGA) gene. Clonal expansion of PIGA-mutated cells occurs uniquely in the setting of bone marrow (BM) failure…
Real-World Effectiveness and Safety of Ravulizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria: Evidence From the International PNH Registry
Ravulizumab, a second-generation complement component 5 inhibitor (C5i) derived from eculizumab eculizumab:
Switching patients with PNH from pegcetacoplan to iptacopan: a case series
Objectives: Limited published data exist on switching therapy from pegcetacoplan pegcetacoplan:
Furha Cossor, MD
Furha Cossor, MD, is a board-certified oncologist and hematologist with Saint Luke's Cancer Specialists. Dr. Cossor has a special interest in multiple myeloma, leukemia, lymphoma, myelodysplastic syndrome, and myeproliferative neoplasms.
Anand B. Karnad, MD
My career since completing my fellowship training in Hematology and Medical Oncology at Boston City Hospital has been devoted to being a clinician-educator passionate about clinical trials as the best option for care. I have led Divisions of Hematology-Oncology at ETSU and at UT Health San Antonio and have played leadership roles in the Cancer Centers at both institutions. My current career focus is on hematology (blood disorders) spanning classical (or benign) hematology and all related pre-malignant and malignant disorders of the blood, bone marrow, lymph nodes, and immune system.
Ludovica Marando, MD
Dr. Marando is a Hematologist in Houston, Texas. Dr. Marando is a dedicated hematologist who is passionate about understanding the mechanisms that govern somatic clonal mosaicism within the hematopoietic stem cell compartment and the factors that govern progression into overt myeloid neoplasms. I do firmly believe that a better understanding of these mechanisms can lead to a shift in treatment paradigms: prevention of deadly hematological cancers.
Brion V. Randolph, MD
Dr. Brion Randolph is a medical oncologist and hematologist with 23 years of experience treating patients with cancer and blood disorders. His expertise encompasses both oncology and hematology, allowing him to provide comprehensive care for patients facing complex diagnoses. Dr. Randolph focuses on delivering personalized treatment plans for individuals dealing with various forms of cancer and blood-related conditions.
Julia T. Warren, MD, PhD
Julia T. Warren, MD, PhD, joined Children's Hospital of Philadelphia in fall 2022. She is a hematologist with the Division of Hematology and the Pediatric Comprehensive Bone Marrow Failure Program.