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While drug discovery for common disorders is somewhat standard and based on the large potential markets, the small market for most orphan diseases means that effective therapies are uncommon. With next generation DNA sequencing, the pace of inherited novel rare disease gene identification has increased markedly. Drug discovery for rare diseases is somewhat hampered by a lack of collaboration between academia and industry separating the necessary resources from skills. This volume discusses important aspects of orphan drug development such as regulatory affairs, public industry partnership, bench to bedside using examples of rare diseases, challenges of clinical trials and future directions.
During the last decades, numerous promising drugs that provided neuroprotection in the laboratory setting failed to translate into the clinic because of their toxicity or lack of efficacy. However, these apparently disappointing results have provided valuable lessons to improve research strategies and enhance their clinical translation. In this scenario, by bringing together the experience developed by stroke researchers and clinicians, the book provides a precious tool for improving stroke patients¿ treatment and management.
This book provides an up-to-date review of the function of microRNA (miRNA) in neurological diseases as well as advancements in technology for therapeutic modulation of miRNA activity. It covers general concepts and novel insights on brain function in relation to miRNA and also examines the role of miRNAs in various neurological diseases. Covering the full range of relevant concepts and giving a look into present and future possibilities, it is a necessary cutting-edge reference for neurologists, neuropathologists, and researchers and practitioners in a wide range of related fields.
During the last decades, numerous promising drugs that provided neuroprotection in the laboratory setting failed to translate into the clinic because of their toxicity or lack of efficacy. However, these apparently disappointing results have provided valuable lessons to improve research strategies and enhance their clinical translation. In this scenario, by bringing together the experience developed by stroke researchers and clinicians, the book provides a precious tool for improving stroke patients' treatment and management.
Understanding and addressing the current opioid crisis requires knowledge of the endogenous opioids (endorphins and enkephalins), but there is now evidence for a benzodiazepine crisis. Are there endogenous benzodiazepine-like substances ¿ and what do they do? How do they affect the anti-anxiety drugs and their adverse effects?
Understanding and addressing the current opioid crisis requires knowledge of the endogenous opioids (endorphins and enkephalins), but there is now evidence for a benzodiazepine crisis. Are there endogenous benzodiazepine-like substances ¿ and what do they do? How do they affect the anti-anxiety drugs and their adverse effects?
Drug Repositioning: Approaches and Applications for Neurotherapeutics reviews history and advances in drug repositioning, with a special focus on therapeutics for nervous system diseases.
This book provides an up-to-date review of the function of microRNA (miRNA) in neurological diseases as well as advancements in technology for therapeutic modulation of miRNA activity. It covers general concepts and novel insights on brain function in relation to miRNA and also examines the role of miRNAs in various neurological diseases. Coveri
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