{"title":"罕见病的临床开发创新:克服尿酸尿随机临床试验成功交付的障碍","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":"{\"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}","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}
Clinical Development Innovation in Rare Diseases: Overcoming Barriers to Successful Delivery of A Randomised Clinical Trial in Alkaptonuria
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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