{"title":"Treating spinal muscular atrophy in the womb","authors":"","doi":"10.1038/d41591-025-00017-9","DOIUrl":null,"url":null,"abstract":"An N-of-1 study suggests that prenatal therapy for spinal muscular atrophy — enabled by transplacental passage of risdiplam from mother to fetus — could help prevent symptom onset before birth.","PeriodicalId":19037,"journal":{"name":"Nature Medicine","volume":"1 1","pages":""},"PeriodicalIF":50.0000,"publicationDate":"2025-03-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Nature Medicine","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1038/d41591-025-00017-9","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"BIOCHEMISTRY & MOLECULAR BIOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
An N-of-1 study suggests that prenatal therapy for spinal muscular atrophy — enabled by transplacental passage of risdiplam from mother to fetus — could help prevent symptom onset before birth.
期刊介绍：
Nature Medicine is a monthly journal publishing original peer-reviewed research in all areas of medicine. The publication focuses on originality, timeliness, interdisciplinary interest, and the impact on improving human health. In addition to research articles, Nature Medicine also publishes commissioned content such as News, Reviews, and Perspectives. This content aims to provide context for the latest advances in translational and clinical research, reaching a wide audience of M.D. and Ph.D. readers. All editorial decisions for the journal are made by a team of full-time professional editors.
Nature Medicine consider all types of clinical research, including:
-Case-reports and small case series
-Clinical trials, whether phase 1, 2, 3 or 4
-Observational studies
-Meta-analyses
-Biomarker studies
-Public and global health studies
Nature Medicine is also committed to facilitating communication between translational and clinical researchers. As such, we consider “hybrid” studies with preclinical and translational findings reported alongside data from clinical studies.
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