AVXS-101基因替代疗法(GRT)治疗1型脊髓性肌萎缩症(SMA1):关键的3期研究(STR1VE)更新

{"title":"AVXS-101基因替代疗法(GRT)治疗1型脊髓性肌萎缩症(SMA1):关键的3期研究(STR1VE)更新","authors":"R. Shell, J. W. Day, C. Chiriboga, Thomas O. Crawford, Basil T. Darras, Richard S. Finkel, Anne M. Connolly, S. Iannaccone, Nancy L Kuntz, L. Pena, M. Schultz, Perry B. Shieh, E. Smith, D. Feltner, F. Ogrinc, Thomas A. Macek, C. Wells, L. Muehring, J. L'italien, D. Sproule, Brian K. Kaspar, J. Mendell","doi":"10.1164/ajrccm-conference.2019.199.1_meetingabstracts.a2789","DOIUrl":null,"url":null,"abstract":"","PeriodicalId":120590,"journal":{"name":"B26. CLINICAL STUDIES IN PEDIATRIC SLEEP, HOME VENTILATION AND NEUROMUSCULAR DISEASE","volume":"107 1","pages":"0"},"PeriodicalIF":0.0000,"publicationDate":"2019-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"1","resultStr":"{\"title\":\"AVXS-101 Gene Replacement Therapy (GRT) for Spinal Muscular Atrophy Type 1 (SMA1): Pivotal Phase 3 Study (STR1VE) Update\",\"authors\":\"R. Shell, J. W. Day, C. Chiriboga, Thomas O. Crawford, Basil T. Darras, Richard S. Finkel, Anne M. Connolly, S. Iannaccone, Nancy L Kuntz, L. Pena, M. Schultz, Perry B. Shieh, E. Smith, D. Feltner, F. Ogrinc, Thomas A. Macek, C. Wells, L. Muehring, J. L'italien, D. Sproule, Brian K. Kaspar, J. Mendell\",\"doi\":\"10.1164/ajrccm-conference.2019.199.1_meetingabstracts.a2789\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"\",\"PeriodicalId\":120590,\"journal\":{\"name\":\"B26. CLINICAL STUDIES IN PEDIATRIC SLEEP, HOME VENTILATION AND NEUROMUSCULAR DISEASE\",\"volume\":\"107 1\",\"pages\":\"0\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2019-05-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"1\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"B26. CLINICAL STUDIES IN PEDIATRIC SLEEP, HOME VENTILATION AND NEUROMUSCULAR DISEASE\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1164/ajrccm-conference.2019.199.1_meetingabstracts.a2789\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"B26. CLINICAL STUDIES IN PEDIATRIC SLEEP, HOME VENTILATION AND NEUROMUSCULAR DISEASE","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1164/ajrccm-conference.2019.199.1_meetingabstracts.a2789","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}