Beta thalassemia (BT) is a rare genetic disorder characterized by the reduced production of hemoglobin. It is caused by a mutation in one hemoglobin beta (HBB) gene (beta thalassemia minor) or a…
Clarivate Epidemiology's coverage of beta thalassemia (BT) comprises epidemiological estimates of key patient populations across the major mature pharmaceutical markets (the United States,…
Approximately 80% of rare diseases have a genetic etiology. Gene therapies hold the potential to transform the treatment of rare diseases and may provide a functional cure for some. However, these…
Beta thalassemia is a rare genetic blood disorder characterized by a substantial reduction in or lack of beta-globin protein, resulting in chronic anemia, failure to thrive, jaundice, pallor, poor…
Renewed interest in the development of gene therapies has fueled commercial interest and investment into novel gene replacement technologies, which has resulted in a strong, diverse gene therapy…
Beta thalassemia is a rare genetic blood disorder characterized by a substantial reduction in or lack of beta-globin protein, resulting in chronic anemia, failure to thrive, jaundice, pallor, poor…
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,…
MARKET OUTLOOK Progress in R&D and technology, regulatory and monetary incentives for advancing the development of treatments for rare diseases, and ongoing focus by the pharmaceutical market on…
MARKET OUTLOOK Progress in R&D and technology, regulatory and monetary incentives for advancing the development of treatments for rare diseases, and ongoing focus by the pharmaceutical market on…
The Orphan Drug Act of 1983 encourages the development of drugs for rare diseases, a process with unique clinical and commercial challenges. Although manufacturers of orphan drugs generally enjoy…
MARKET OUTLOOK Progress in R&D and technology, regulatory and monetary incentives for advancing the development of treatments for rare diseases, and ongoing focus by the pharmaceutical market on…
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…
Nearly 35 years after the Orphan Drug Act of 1983 (ODA) became law in the United States, private investment in the development of drugs for orphan diseases continues unabated. Although the growing…
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…