Paroxysmal Nocturnal Hemoglobinuria (PNH)

Advances in Paroxysmal Nocturnal Hemoglobinuria, AAMDSIF 2026 International Bone Marrow Failure Scientific Symposium

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Topic(s)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Presenter(s)
Austin Kulasekararaj, MBBS, MD
Pedro Prata, MD, PhD
Regis Peffault de Latour, MD, PhD
Bruno Fattizzo, MD
Morag Griffin, MBChB, FRCPath
Jun Ho Jang, MD, PhD
David Araten, MD

Recording from the AAMDSIF 2026 International Bone Marrow Failure Scientific Symposium, Co-Chairs Austin Kulasekarararaj, MBBS, and Pedro Prata, MD, PhD PNH Updates for 2026 - Regis Peffault de LaTour, MD, PhD Recognition and management of breakthrough hemolysis - Bruno Fattizzo…

Good job, Good health, Good life. Then: Aplastic Anemia, Followed by PNH. Daniel’s Story

In 2009, Daniel had a good job in accounting in Sydney, Australia. He had a structured, organized, healthy lifestyle. He had a devoted partner (whom he later married) and was studying for the Chartered Accountancy qualification. He rode his bike to work and played soccer. He was planning a good life ahead.

One day at the office, the receptionist pinched his arm. The next day, he saw a huge welt in the same spot.  Because a colleague urged him to get it checked out, he did.

Health-Related Quality of Life, AAMDSIF 2026 International Bone Marrow Failure Scientific Symposium

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Presenter(s)
Namrata Chandhok, MD
Thomas LeBlanc, MD, MA
Fabio Efficace, PhD
Alison R. Kavanaugh
Pamela C. Egan, MD
Wil L. Santivasi, MD
The session was recorded at the AAMDSIF 2026 International Bone Marrow Failure Scientific Symposium, Co-Chairs Namrata Chandhok, MD, and Thomas LeBlanc, MD, MA Topics and speakers included: How can we measure quality of life in MDS, to inform interventions? - Fabio Efficace, PhD…

Non-Transplant Therapies for Marrow Failure, AAMDSIF 2026 International Bone Marrow Failure Scientific Symposium

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Presenter(s)
Carlos DeCastro, MD
Phil Scheinberg, MD
Regis Peffault de Latour, MD, PhD
Julian Sevilla, MD, PhD
Kasiani Myers, MD
Suneet Agarwal, MD, PhD
This session was presented at the AAMDSIF 2026 International Bone Marrow Failure Scientific Symposium; Co-Chairs Carlos DeCastro, MD, and Phil Scheinberg, MD, PhD. Session topics included: -Androgens, alemtuzumab, anti-cytokine therapies (anti-IL2R, anti-IFN gamma), daratumumab…

Clonality in Acquired Bone Marrow Failure, PNH, and VEXAS, AAMDSIF 2026 International Bone Marrow Failure Scientific Symposium

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Topic(s)
Aplastic Anemia
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Presenter(s)
Emma Groarke, MD, FRCPath
Yoshitaka Zaimoku, MD, PhD
Hiroki Mizumaki, MD
Martina Fiumara, PhD
Austin Kulasekararaj, MBBS, MD
Bhavisha Patel, MD
Thibault Comont, MD
This session, Clonality in Acquired Bone Marrow Failure, PNH, and VEXAS, was recorded at the AAMDSIF 2026 International Scientific Symposium; session Co-Chairs Emma Groarke, MD, and Yoshitaka Zaimoku, MD, PhD Session topics and speakers included: -Insights of PNH using single…

David Young, MD, PhD

Institution
NIH
Primary Disease Area of Focus
Aplastic Anemia
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Pediatric
About

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

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