Until My Son Calls Me Dad: Quest to Find a Treatment for Ultra-Rare Disease.

IF 2.4 4区医学 Q3 PHARMACOLOGY & PHARMACY

Journal of clinical pharmacology Pub Date : 2022-12-01 DOI:10.1002/jcph.2154

Sanath Kumar Ramesh

引用次数: 1

Abstract

Rare diseases are affecting 400 million patients worldwide, with 95% of them suffering without treatments. In this article, I make a plea, as a parent of a rare disease kid, and as a drug developer, to turn the attention of pharmacologists to such rare and devastating diseases.

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直到我儿子叫我爸爸:寻找一种治疗超罕见疾病的方法。

全世界有4亿患者患有罕见病，其中95%的患者得不到治疗。在这篇文章中，作为一个患有罕见疾病的孩子的父母，作为一个药物开发者，我恳请药理学家把注意力转向这种罕见的、毁灭性的疾病。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Journal of clinical pharmacology 医学-药学

CiteScore

5.10

自引率

3.40%

发文量

176

审稿时长

2 months

期刊介绍： The Journal of Clinical Pharmacology (JCP) is a Human Pharmacology journal designed to provide physicians, pharmacists, research scientists, regulatory scientists, drug developers and academic colleagues a forum to present research in all aspects of Clinical Pharmacology. This includes original research in pharmacokinetics, pharmacogenetics/pharmacogenomics, pharmacometrics, physiologic based pharmacokinetic modeling, drug interactions, therapeutic drug monitoring, regulatory sciences (including unique methods of data analysis), special population studies, drug development, pharmacovigilance, womens’ health, pediatric pharmacology, and pharmacodynamics. Additionally, JCP publishes review articles, commentaries and educational manuscripts. The Journal also serves as an instrument to disseminate Public Policy statements from the American College of Clinical Pharmacology.