Rare Diseases and Orphan Drugs: Keys to Understanding and Treating the Common Diseases Kindle Edition
Author: Visit ‘s Jules J. Berman Page ID: B00LBXYKVU
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File Size: 2908 KBPrint Length: 400 pagesPublisher: Academic Press; 1 edition (May 26, 2014)Publication Date: May 26, 2014 Sold by: Digital Services, Inc. Language: EnglishID: B00LBXYKVUText-to-Speech: Enabled X-Ray: Not Enabled Word Wise: Not EnabledLending: Not Enabled Enhanced Typesetting: Not Enabled Best Sellers Rank: #1,705,589 Paid in Kindle Store (See Top 100 Paid in Kindle Store) #522 in Kindle Store > Kindle eBooks > Medical eBooks > Research #2084 in Kindle Store > Kindle eBooks > Medical eBooks > Diseases #2516 in Books > Medical Books > Research
This is a fascinating and important book. As a physician, I have always taught my students and residents that they should not devote much time or effort toward learning about or researching rare diseases. My justification? Too little bang for the buck- no matter what you discover about a rare tumor, it will have virtually no impact compared to discovering something about, say, breast or lung cancer.
Well, I was wrong about this, and Dr. Berman, in “Rare diseases and orphan drugs,” clearly demonstrates why this is completely fallacious. In fact, studying rare diseases has provided incredibly more insight and more new drugs than the study of common diseases. The reasons for this and many other related but often counterintuitive concepts are boiled down to a set of rules. Importantly, he points out that “…rare diseases are not the exceptions to the general rules that apply to common diseases; the rare diseases are the exceptions upon which the general rules of common diseases are based.” He tells us that “every rare disease tells us something about the normal functions of organisms.” He points out the well-known fact that that the diseases that cause the most deaths receive the most funding, and attributes this to the “simple minded expectation” that this will provide the greatest societal benefit. Why this expectation is wrong is one of the main purposes of the book and is highlighted throughout.
Only a basic understanding of biology is needed to appreciate this book. Berman often makes concepts very clear using well-chosen analogies from manufacturing to fingerpainting. He often takes a very high overview of biological life from single cells to plants and animals, identifying and tying together general principles of cellular life in ways that I have never imagined.
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