Defining rare conditions in the era of personalized medicine

被引：1
|
作者：
O'Connor, Daniel J. [1 ,2 ]
Gabaldo, Michela [1 ,3 ]
Aartsma-Rus, Annemieke [1 ,4 ,5 ]
Jonker, Anneliene Hechtelt [1 ,6 ]
机构：
[1] Therapies Sci Comm, Int Rare Dis Res Consortium, Paris, France
[2] Assoc British Pharmaceut Ind, London, England
[3] Aptuit Evotec Co, Verona, Italy
[4] Leiden Univ, Med Ctr, Dept Human Genet, Leiden, Netherlands
[5] Dutch Ctr RNA Therapeut, Leiden, Netherlands
[6] Univ Twente, Enschede, Netherlands
来源：
NATURE REVIEWS DRUG DISCOVERY | 2023年 / 22卷 / 11期
关键词：
D O I：
10.1038/d41573-023-00145-2
中图分类号：
Q81 [生物工程学（生物技术）]; Q93 [微生物学];
学科分类号：
071005 ; 0836 ; 090102 ; 100705 ;
摘要：
The total number of rare conditions is debated, partly because of the variety of definitions of what constitutes rare. A broader consensus view of what rare means, based on improved understanding of individual group and patient clinicopathological characteristics, will help maximize the impact of technological advances in therapeutic development programmes.
引用
收藏
页码：857 / 858
页数：2
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