Extending the interval between pegunigalsidase alfa infusions in patients with Fabry disease: Five-year interim results from the ongoing BRIGHT51 study

{"title":"Extending the interval between pegunigalsidase alfa infusions in patients with Fabry disease: Five-year interim results from the ongoing BRIGHT51 study","authors":"John A. Bernat , Myrl Holida , Stephen Waldek , William R. Wilcox , Nicola Longo , Ozlem Goker-Alpan , Eric Wallace , Patrick Deegan , Camilla Tøndel , Francois Eyskens , Ulla Feldt-Rasmussen , Derralynn Hughes , Antonio Pisani , Ankit Mehta , Khan Nedd , David G. Warnock , Giovanni Piotti , Meng Wang , Einat Almon-Brill , Sari Alon , Aleš Linhart","doi":"10.1016/j.ymgme.2024.108656","DOIUrl":null,"url":null,"abstract":"","PeriodicalId":18937,"journal":{"name":"Molecular genetics and metabolism","volume":"144 2","pages":"Article 108656"},"PeriodicalIF":3.7000,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Molecular genetics and metabolism","FirstCategoryId":"99","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S1096719224005407","RegionNum":2,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"ENDOCRINOLOGY & METABOLISM","Score":null,"Total":0}