About the book
A rare disease is any disease or condition that affects a small percentage of the population. In the United States alone, over 7000 rare diseases affect more than thirty million people. Many rare conditions are not only life-threatening or chronically debilitating diseases but most do not have appropriate treatments, rendering them incurable. Consequently, drug, biologic, and device development in the case of rare diseases is challenging for many reasons, including the complex biology and the lack of critical understanding of the natural course of many rare diseases. In addition, the inherently fewer population of patients with a rare disease can also make conducting clinical trials difficult. Scientists and clinicians around the globe are working to find better ways to prevent, detect, and treat a rare disease, and to improve the quality of life of patients. In recent years, there is a substantial amount of development in the area of rare disease research and its clinical applications, for instance, rare disease biology and genomics, epidemiology and preventions; early detection and screening; as well as diagnosis and treatment. Besides, since the advent of various emerging technologies, such as genome editing technology, stem cell technology, bionanotechnology, as well as regenerative medicine-and the integrated knowledge gained from such studies, not only enhancing our understanding of rare disease but also producing novel insights that could lead to the development and timely deployment of novel clinical/therapeutic interventions.
In this context, the objective of this book will be to consolidate the recent advances in the area of rare disease biology/therapeutics covering a wide spectrum of interrelated topics in a timely fashion, and to disseminate that essential knowledge in a comprehensible way to a greater scientific and clinical audience; as well as patients, caregivers, and drug and device manufacture, especially to support rare disease product development.