Disease Landscape and Forecast Insights

Myelodysplastic syndromes (MDS) are a heterogeneous collection of hematopoietic stem cell disorders that result in cytopenias and the risk of progression to acute myeloid leukemia. Treatment goals…

Sickle cell disease (SCD) is a rare genetic blood disorder characterized by polymerization of hemoglobin in red blood cells (RBCs) that distorts them into a sickle shape. This sickling leads to…

MARKET OUTLOOKSuccessive label expansions in the multiple myeloma treatment landscape are driving significant commercial reward for some well-entrenched current therapies. With Celgene’s Revlimid…

Beta thalassemia is a rare genetic blood disorder characterized by a substantial reduction in or lack of β-globin protein, which results in chronic anemia, failure to thrive, jaundice, pallor,…

Polycythemia Vera (PV) is a rare hematological malignancy estimated to affect approximately 50 thousand patients in the United States. Based on patient’s age and prior clinical history patients…

Immune thrombocytopenic purpura (ITP) is a hematological autoimmune disorder characterized by low platelet count in the blood, which may lead to easy bruising and bleeding. ITP has an acute,…

Beta thalassemia is a rare genetic blood disorder characterized by a substantial reduction in or lack of β-globin protein and resulting in chronic anemia, failure to thrive, jaundice, pallor, poor…

The chronic lymphocytic leukemia (CLL) and chronic myeloid leukemia (CML) markets are becoming increasingly crowded, with recent marketing authorizations expanding therapy options for patients. As…

Sickle cell disease (SCD) is a genetic disorder characterized by abnormality in the oxygen-carrying protein hemoglobin. The gene mutation underlying the disease results in the amino acid valine…

Beta thalassemia is a recessive monogenic blood disorder characterized by low levels or an absence of the protein ß-globin, a key component of the oxygen transporting protein hemoglobin. A lack of…

Hemophilia is a monogenic bleeding disorder occurring primarily in males. In hemophilia A and hemophilia B, factor VIII or factor IX (respectively) is either absent or deficient in function…

Mutations in the gene coding for C1-inhibitor (C1-INH) protein result in hereditary angioedema (HAE), whereby insufficient levels of C1-INH or abnormal forms of the protein ultimately result in…