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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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发表于 2025-3-21 18:45:26 | 显示全部楼层 |阅读模式
书目名称Rare Disease Drug Development
副标题Clinical, Scientific
编辑Raymond A. Huml
视频videohttp://file.papertrans.cn/822/821231/821231.mp4
概述Provides patient prospective and narratives.Addresses relevant operational and developmental processes in drug development.Provides comments from prominent CEOs in the biopharmaceutical and rare disea
图书封面Titlebook: Rare Disease Drug Development; Clinical, Scientific Raymond A. Huml Book 2021 The Editor(s) (if applicable) and The Author(s), under exclus
描述.This book provides a broad overview of rare disease drug development. It offers unique insights from various perspectives, including third-party capital providers, caregivers, patient advocacy groups, drug development professionals, marketing and commercial experts, and patients. ..A unique reference, the book begins with narratives on the many challenges faced by rare disease patient and their caregivers. Subsequent chapters underscore the critical, multidimensional role of patient advocacy groups and the novel approaches to related clinical trials, investment decisions, and the optimization of rare disease registries. The book addresses various rare disease drug development processes by disciplines such as oncology, hematology, pediatrics, and gene therapy. Chapters then address the operational aspects of drug development, including approval processes, development accelerations, and market access strategies. The book concludes with reflections on the authors‘ case forreal-world data and evidence generation in orphan medicinal drug development. ..Rare Disease Drug Development. is an expertly written text optimized for biopharmaceutical R&D experts, commercial experts, third-party
出版日期Book 2021
关键词Rare disease; drug development; caregivers; orphan drug; disease-modifying treatments
版次1
doihttps://doi.org/10.1007/978-3-030-78605-2
isbn_softcover978-3-030-78607-6
isbn_ebook978-3-030-78605-2
copyrightThe Editor(s) (if applicable) and The Author(s), under exclusive license to Springer Nature Switzerl
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发表于 2025-3-21 21:51:22 | 显示全部楼层
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发表于 2025-3-22 03:55:07 | 显示全部楼层
https://doi.org/10.1007/978-3-030-78605-2Rare disease; drug development; caregivers; orphan drug; disease-modifying treatments
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Introduction to Rare Diseases and Market Overview, and attempt to identify approaches to target those mechanisms to modify or perhaps cure the disease. Only a small number of rare diseases had approved therapies..Since then, there has been a marked increase in rare disease research funding and development effort, thanks to regulatory changes, multi
发表于 2025-3-22 17:13:47 | 显示全部楼层
Select Patient Narratives,nto the rare disease drug development and approval processes. Rare disease patients are the most appropriate individuals to consult in order to identify and rank the importance of the conditions that they face..This chapter will provide multiple views from persons/patients afflicted with different r
发表于 2025-3-22 22:04:53 | 显示全部楼层
A Mental Health Perspective,hapter will define terms related to mental health, provide an overview of the current state of mental health in the rare disease population, discuss the gaps and unmet needs that have been identified, and offer considerations for strategies and measures to address the gaps in providing mental health
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