Paroxysmal Nocturnal Hemoglobinuria (PNH)
The Path to Accessible Care: Development and Impact of Eculizumab Biosimilars for Paroxysmal Nocturnal Hemoglobinuria and Atypical Hemolytic Uremic Syndrome
Eculizumab Eculizumab: Eculizumab (Soliris ®) is given as an IV into a vein at the doctor’s office or at a special center. The procedure usually takes about 35 minutes.
Urge your Members of Congress to Reverse the 57% Cut to the Congressionally Directed Medical Research Program
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Take Action
Federal budget negotiations are already under way. April 15 marks the 30-Day anniversary of the enactment of the 57% cut to CDMRP including the elimination of all funding to Bone Marrow Failure Research. Every medical research program at CDMRP directly impacts the health and…
AAMDSIF Supports the Accelerating Kids' Access to Care Act
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Coalition Action
AAMDSIF Supports the Accelerating Kids' Access to Care Act. On March 26, 2025, AAMDSIF joined with 212 additional patient advocacy and medical center organizations in urging members of the US Congress to pass the Accelerating Kids’ Access to Care Act. Passage of this bill will…
How Our Lungs Back Up the Bone Marrow to Make Our Blood
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A study of human lung samples reveals a potent new source of hematopoietic stem cells, which make red blood cells, platelets and immune cells. By Levi Gadye Red blood cells carry oxygen from the lungs to every other organ, and blood-forming stem cells must make about 200 billion…
2025 Rockville Patient and Family Conference Session: PNH
Topic(s)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Presenter(s)
Bhumika Patel, MD
In this recording, Dr. Bhumika Patel discusses what is new in PNH and current treatment options available to patients.
2025 Rockville Patient and Family Conference: PNH Complications with Philip Scheinberg
Topic(s)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Presenter(s)
Phil Scheinberg, MD
In this recording, Dr. Philip Scheinberg discusses PNH complications that can impact a patient - extravascular hemolysis, intravascular hemolysis, Budd-Chiari Syndrome and blood clots.
2025 AAMDSIF Bone Marrow Failure Summit Paroxysmal Nocturnal Hemoglobinuria Expert Panel
Topic(s)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Presenter(s)
Regis Peffault de Latour, MD, PhD
Austin Kulasekararaj, MBBS, MD
Morag Griffin, MBChB, FRCPath
Carlos M. De Castro, III, M.D.
Watch PNH experts at the 2025 AAMDSIF Bone Marrow Failure Summit tackle the latest science and research on choosing the optimal complement inhibition strategy in PNH. This session is moderated by Phillip Scheinberg, MD from Hopital A Beneficencia in Sao Paulo, Brazil. Session…
Thrombosis at Unusual Sites: Focus on Myeloproliferative Neoplasms and Paroxysmal Nocturnal Hemoglobinuria
Patients with thrombosis thrombosis: (throm-BOE-suss) A blood clot (thrombus) that develops and attaches to a blood vessel. at an unusual site will need to be explored for rare causes of