{"title":"脊髓性肌萎缩症目前和新出现的治疗方案。","authors":"Faraz Farooq, Alex E MacKenzie","doi":"10.2147/DNND.S48420","DOIUrl":null,"url":null,"abstract":"<p><p>Spinal muscular atrophy is one of the most common inherited neuromuscular conditions; our understanding of the genetic pathology and translational research coming from this insight has made significant progress over the past decade. This short review provides the background of the disease along with the bench to bedside progress of some promising treatment options to develop better understanding of the present state of the disease.</p>","PeriodicalId":11147,"journal":{"name":"Degenerative Neurological and Neuromuscular Disease","volume":"5 ","pages":"75-81"},"PeriodicalIF":0.0000,"publicationDate":"2015-07-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/DNND.S48420","citationCount":"0","resultStr":"{\"title\":\"Current and emerging treatment options for spinal muscular atrophy.\",\"authors\":\"Faraz Farooq, Alex E MacKenzie\",\"doi\":\"10.2147/DNND.S48420\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Spinal muscular atrophy is one of the most common inherited neuromuscular conditions; our understanding of the genetic pathology and translational research coming from this insight has made significant progress over the past decade. This short review provides the background of the disease along with the bench to bedside progress of some promising treatment options to develop better understanding of the present state of the disease.</p>\",\"PeriodicalId\":11147,\"journal\":{\"name\":\"Degenerative Neurological and Neuromuscular Disease\",\"volume\":\"5 \",\"pages\":\"75-81\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2015-07-17\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://sci-hub-pdf.com/10.2147/DNND.S48420\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Degenerative Neurological and Neuromuscular Disease\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.2147/DNND.S48420\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2015/1/1 0:00:00\",\"PubModel\":\"eCollection\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Degenerative Neurological and Neuromuscular Disease","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.2147/DNND.S48420","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2015/1/1 0:00:00","PubModel":"eCollection","JCR":"","JCRName":"","Score":null,"Total":0}
Current and emerging treatment options for spinal muscular atrophy.
Spinal muscular atrophy is one of the most common inherited neuromuscular conditions; our understanding of the genetic pathology and translational research coming from this insight has made significant progress over the past decade. This short review provides the background of the disease along with the bench to bedside progress of some promising treatment options to develop better understanding of the present state of the disease.
求助内容：
应助结果提醒方式：
京公网安备 11010802042870号
