{"title":"Clinical Development Innovation in Rare Diseases: Overcoming Barriers to Successful Delivery of A Randomised Clinical Trial in Alkaptonuria","authors":"Ranganath Lr, Sireau N","doi":"10.53043/ndcr-2-008","DOIUrl":null,"url":null,"abstract":"Alkaptonuria (AKU) (OMIM#203500) is a rare inherited disorder due to deficiency of homogentisate dioxygenase with resultant accumulation of homogentisic acid, occurring with a worldwide frequency of 1 in 100,000 to 250,000 [1].","PeriodicalId":330764,"journal":{"name":"Journal of Clinical Medicine: Current Research","volume":"76 1","pages":"0"},"PeriodicalIF":0.0000,"publicationDate":"2022-08-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Clinical Medicine: Current Research","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.53043/ndcr-2-008","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 0
Abstract
Alkaptonuria (AKU) (OMIM#203500) is a rare inherited disorder due to deficiency of homogentisate dioxygenase with resultant accumulation of homogentisic acid, occurring with a worldwide frequency of 1 in 100,000 to 250,000 [1].
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