{"title":"Until My Son Calls Me Dad: Quest to Find a Treatment for Ultra-Rare Disease.","authors":"Sanath Kumar Ramesh","doi":"10.1002/jcph.2154","DOIUrl":null,"url":null,"abstract":"<p><p>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.</p>","PeriodicalId":2,"journal":{"name":"ACS Applied Bio Materials","volume":null,"pages":null},"PeriodicalIF":4.6000,"publicationDate":"2022-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"1","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"ACS Applied Bio Materials","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1002/jcph.2154","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"MATERIALS SCIENCE, BIOMATERIALS","Score":null,"Total":0}
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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