Rare Diseases and Orphan Drugs

Rare Hematological Diseases

When the normal process of blood cell growth and development breaks down, certain cell types can be produced in abnormally large quantities or in abnormal form which gives rise to blood cancers or other blood disorders. There are two types of rare blood diseases, they include: Myeloproliferative disorders also referred as myeloproliferative neoplasms (MPNs) and Lymph proliferative disorders also referred as lymph proliferative neoplasms (LPNs). Myeloproliferative disorders originate when the bone marrow starts making too many red blood cells, platelets, or certain white blood cells whereas, lymph proliferative disorders.MPNs include: Polycythemia Vera, Myelofibrosis, Essential thrombocythemia, Eosinophilia, Mastocytosis, Histiocytosis and Paroxysmal nocturnal hemoglobinuria. LPNs include: Hairy cell leukemia (HCL) and Large Granular Leukemia (LGL).