Rare Diseases and Orphan Drugs

Rare Haematology Diseases

Hematologic diseases, disorders of the blood and blood-forming organs, afflict many Americans. In addition to blood corpuscle cancers, hematologic diseases include rare genetic disorders, anaemia, conditions associated with HIV, red blood cell disease, and complications from chemotherapy or transfusions.NIDDK-supported haematology researchers work in many various areas, from developing drugs to support people that receive multiple blood transfusions, to laboratory research to raised understand the traditional and abnormal function of blood cells. The NIDDK also supports research within the biology of adult blood stem cells, which are needed for bone marrow transplants and should have broader application in gene therapy research.

• Acquired hemophilia
• Acquired hemophilia A
• Acquired pure red cell aplasia
• Acquired Von Willebrand syndrome
• Acute erythroid leukemia
• Acute graft versus host disease
• Acute monoblastic leukemia

Related Associations of Rare Diseases Research:

Alzheimer's disease Organizations, Ann & Robert H. Lurie Children’s Hospital, Birmingham children’s Hospital, Boston Children’s Hospital, Canadian Organization for Rare Diseases, Chicago Rare Disease Foundation, Children’s hospital of Pittsburgh, National Alliances for Rare Diseases, National Institute of Health (NIH), National Organization for Rare Diseases, NDC Medicine, Rare Diseases Patient Association Funding, Rare Diseases South Africa, Rare Diseases Translational Research Collaboration.