Titlebook: Rare Disease Drug Development; Clinical, Scientific Raymond A. Huml Book 2021 The Editor(s) (if applicable) and The Author(s), under exclus

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Introduction to Rare Diseases and Market Overview,ercent have exclusively pediatric onset), affecting 3–4 percent of all births. Although the number of individuals suffering from a single rare disease is by definition small, the total number of individuals that rare diseases impact is significant, at approximately 25 million people in the United St

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The Patient Perspective,as passive “subjects” and consumers, patients today demand to have a voice and role in the therapeutic development process, from clinical outcomes and the design of clinical trials to the assessment of risk benefit and, ultimately, access to therapies. This is especially true in the rare disease spa