Research is key to understanding, treating, and ultimately finding cures for paroxysmal nocturnal hemoglobinuria (PNH) and other bone marrow failure diseases. We know that many patients, family members, and healthcare professionals follow research developments very closely and frequently. This section of the website provides information on PNH research, much of it funded by the PNH Foundation. To stay up-to-date on the latest research, you can also sign up to receive the monthly electronic newsletter, Research Connection, as well as the PNH Connection, published three times per year from our partner organization, the Aplastic Anemia & MDS International Foundation.
We hope this information is helpful and encouraging to you as we continue to work to provide answers, support, and hope to all who are touched by PNH and bone marrow failure disease.
Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect, or treat disease. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study.
Learn more about the different types of clinical trials and things to consider when deciding whether a clinical trial is a good option for you.
You can also learn more about clinical trials and children.
PNH Research & Support Foundation Research Grants
- Patrizia Ricci, PhD: Small peptide C3-inhibitors for the treatment of paroxysmal nocturnal hemoglobinuria: moving towards the bedside
- Chao-Yie Yang, PhD: Discovery of small molecule complement inhibitors as the treatment for PNH
- Hideki Makishima, MD, PhD: Clonal Architecture in PNH: Somatic genetic defects facilitating clonal expansion
- David Araten, MD: Secondary mutations and thrombosis in PNH
- Jeffrey Pu, MD, PhD: Relevance of PIG-A mutations in acquired bone marrow failure diseases
- Keith R. MacCrae, MD: Circulating microparticles in PNH
- Regis Peffault deLatour, MD, PhD: The role of Unfolded Protein Response in PNH
Reports from Scientific Symposia - lay language explanations of scientific meetings and symposia
2014 AA&MDSIF Scientific Symposium
Highlights from American Society of Hematology (ASH) Annual Meeting 2013
- Complement blockade with a c1 esterase inhibitor in PNH
- PNH: Insights into natural history of PNH
- Dr. Jeffrey Pu of the Penn State Hershey Cancer Institute and an AA&MDSIF Grantee, discusses his research about bone marrow failure disease and explains translational research.
PNH Research Presented at EHA the European Hematology Association annual meeting in June 2013
- Improved Fatigue and Quality-of-Life in Patients with Paroxysmal Nocturnal Hemoglobinuria During Treatment with Eculizumab: Data from the Global PNH Registry
- Eculizumab Protects Against TE and Prolongs Survival in Patients with Paroxysmal Nocturnal Hemoglobinuria: An International PNH Registry Study
PNH Highlights from the 2012 American Society of Hematology Annual Meeting (ASH)
- Eculizumab in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Report of All 153 Patients Treated in the UK
- Genetic Polymorphism of the Complement Receptor-1 (CR1) Gene Correlates with the Clinical Response to Eculizumab of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Novel Complement Modulators for Paroxysmal Nocturnal Hemoglobinuria: Peptide and Protein Inhibitors of C3 Convertase Prevent Both Surface C3 Deposition and Subsequent Hemolysis of Affected Erythrocytes in Vitro
- A Rare Genetic Polymorphism in C5 Confers Poor Response to the Anti-C5 Monoclonal Antibody Eculizumab by Nine Japanese Patients with PNH
- Autoreactive T Cells From Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Specifically Recognize Glycosyl- Phosphatidyl-Inositol (GPI)
Highlights from the 2011 American Society of Hematology Annual Meeting (ASH)
- A Summary of Abstracts for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) and their Caregivers (also available in print)
- Watch interviews from leading experts about the most important information presented at the American Society of Hematology Annual Meeting. Interviews conducted at ASH 2011 by our educational partner, Patient Power.
Highlights from the 2010 American Society of Hematology Annual Meeting
- A Summary of Abstracts for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) and their Caregivers
Research Reviews for Patients
- Bone marrow failure disease reviews in lay language, focused on important medical journal articles on bone marrow failure disease. These reviews are written by hematology/oncology professionals who interpret the most relevant information for patients from the latest published research.