| 书目名称 | Rare Diseases Epidemiology: Update and Overview |
| 编辑 | Manuel Posada de la Paz,Domenica Taruscio,Stephen |
| 视频video | |
| 概述 | Contributors and Authors with Unique Experiences in Rare Diseases Research and Orphan Products Development.Contributors include leading patient advocates, foundation leaders, research investigators, r |
| 丛书名称 | Advances in Experimental Medicine and Biology |
| 图书封面 |  |
| 描述 | .The fields of rare diseases research and orphan products development continue to expand with more products in research and development status. In recent years, the role of the patient advocacy groups has evolved into a research partner with the academic research community and the bio-pharmaceutical industry. Unique approaches to research and development require epidemiological data not previously available to assist in protocol study design and patient recruitment for clinical trials required by regulatory agencies prior to approval for access by patents and practicing physicians.. |
| 出版日期 | Book 2017Latest edition |
| 关键词 | epidemiology; genotype-phenotype correlation; next generation sequencing; orphan drugs; rare diseases |
| 版次 | 2 |
| doi | https://doi.org/10.1007/978-3-319-67144-4 |
| isbn_softcover | 978-3-319-88385-4 |
| isbn_ebook | 978-3-319-67144-4Series ISSN 0065-2598 Series E-ISSN 2214-8019 |
| issn_series | 0065-2598 |
| copyright | This is a U.S. government work and not under copyright protection in the U.S.; foreign copyright pro |
发表于 2025-3-21 17:19:38
