Priya S. Kishnani, Marguerite C. Weinert, James Davison, A. Brassier, Alexander Broomfield, Yin-Hsiu Chien, S. Hahn, D. Kronn, Satoko Kumada, François Labarthe, Hirotaka Ohki, K. Haack, O. Huynh-Ba, S. Sparks, S. Tammireddy, Tianyue Zhou, S. Prakalapakorn
{"title":"使用新型测量设备客观评估小儿庞贝氏症(IOPD)患者上睑下垂的临床疗效:曾接受过阿糖苷酶α治疗的 Mini-COMET 参与者服用阿瓦糖苷酶α 145 周的结果","authors":"Priya S. Kishnani, Marguerite C. Weinert, James Davison, A. Brassier, Alexander Broomfield, Yin-Hsiu Chien, S. Hahn, D. Kronn, Satoko Kumada, François Labarthe, Hirotaka Ohki, K. Haack, O. Huynh-Ba, S. Sparks, S. Tammireddy, Tianyue Zhou, S. Prakalapakorn","doi":"10.1016/j.ymgme.2023.107918","DOIUrl":null,"url":null,"abstract":"","PeriodicalId":504050,"journal":{"name":"Molecular Genetics and Metabolism","volume":"1358 ","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Using a novel measuring device to objectively evaluate the clinical benefit on ptosis in infantile-onset Pompe disease (IOPD): Results from 145 weeks' avalglucosidase alfa dosing in Mini-COMET participants previously treated with alglucosidase alfa\",\"authors\":\"Priya S. Kishnani, Marguerite C. Weinert, James Davison, A. Brassier, Alexander Broomfield, Yin-Hsiu Chien, S. Hahn, D. Kronn, Satoko Kumada, François Labarthe, Hirotaka Ohki, K. Haack, O. Huynh-Ba, S. Sparks, S. Tammireddy, Tianyue Zhou, S. Prakalapakorn\",\"doi\":\"10.1016/j.ymgme.2023.107918\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"\",\"PeriodicalId\":504050,\"journal\":{\"name\":\"Molecular Genetics and Metabolism\",\"volume\":\"1358 \",\"pages\":\"\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2024-02-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Molecular Genetics and Metabolism\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1016/j.ymgme.2023.107918\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Molecular Genetics and Metabolism","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1016/j.ymgme.2023.107918","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
Using a novel measuring device to objectively evaluate the clinical benefit on ptosis in infantile-onset Pompe disease (IOPD): Results from 145 weeks' avalglucosidase alfa dosing in Mini-COMET participants previously treated with alglucosidase alfa