{"title":"Valoctocogene roxaparvovec (Roctavian) - a gene therapy for severe hemophilia A.","authors":"","doi":"10.58347/tml.2023.1686c","DOIUrl":null,"url":null,"abstract":"Valoctocogene roxaparvovec-rvox (Roctavian – Biomarin), an adeno-associated virus (AAV) vector-based gene therapy, has been approved by the FDA for one-time treatment of severe hemophilia A in adults without pre-existing antibodies to AAV serotype 5. It is the first gene therapy to be approved in the US for treatment of hemophilia A.","PeriodicalId":54742,"journal":{"name":"Medical Letter on Drugs and Therapeutics","volume":"65 1686","pages":"157-158"},"PeriodicalIF":1.4000,"publicationDate":"2023-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Medical Letter on Drugs and Therapeutics","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.58347/tml.2023.1686c","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
引用次数: 0
Abstract
Valoctocogene roxaparvovec-rvox (Roctavian – Biomarin), an adeno-associated virus (AAV) vector-based gene therapy, has been approved by the FDA for one-time treatment of severe hemophilia A in adults without pre-existing antibodies to AAV serotype 5. It is the first gene therapy to be approved in the US for treatment of hemophilia A.
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