PNH Research

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 Trials

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

2014 Grantees

  • 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

2013 Grantee

2012 Grantees

2011 Grantee

2009 Grantee

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

PNH Research Presented at EHA the European Hematology Association annual meeting in June 2013

PNH Highlights from the 2012 American Society of Hematology Annual Meeting (ASH)

Highlights from the 2011 American Society of Hematology Annual Meeting (ASH)

Highlights from the 2010 American Society of Hematology Annual Meeting

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.