Rare Diseases and Orphan Drugs - 9780124199880

Rare Diseases and Orphan DrugsKeys to Understanding and Treating the Common Diseases\nAuthor(s): Jules J. Berman\nFormat: Hardback\nPublisher: Elsevier Science Publishing Co Inc, United States\nImprint: Academic Press Inc\nISBN-13: 9780124199880, 978-0124199880\nSynopsis\nRare Diseases and Orphan Drugs shows that much of what we now know about common diseases has been achieved by studying rare diseases. It proposes that future advances in the prevention, diagnosis, and treatment of common diseases will come as a consequence of our accelerating progress in the field of rare diseases. Understanding the complex steps in the development of common diseases, such as cancer, cardiovascular disease, and metabolic diseases, has proven a difficult problem. Rare diseases, however, are often caused by aberrations of a single gene. In rare diseases, we may study how specific genetic defects can trigger a series of events that lead to the expression of a particular disease. Often, the disease pro.