首页> Neurology and therapy> Parent Experiences of Sanfilippo Syndrome Impact and Unmet Treatment Needs: A Qualitative Assessment.

Parent Experiences of Sanfilippo Syndrome Impact and Unmet Treatment Needs: A Qualitative Assessment.

导出 LinkOut

翻译打印收藏纠错

第一作者： Katherine Ackerman,Porter

第一单位： Center for Genomics, Bioinformatics, and Translational Research, RTI International, Research Triangle Park, NC, USA. kmporter@rti.org.

作者： Katherine Ackerman,Porter ^[1] ; Cara,O'Neill ^[2] ; Elise,Drake ^[2] ; Samantha,Parker ^[3] ; Maria L,Escolar ^[4] ; Stacey,Montgomery ^[5] ; William,Moon ^[5] ; Carolyn,Worrall ^[5] ; Holly L,Peay ^[6]

作者单位： Center for Genomics, Bioinformatics, and Translational Research, RTI International, Research Triangle Park, NC, USA. kmporter@rti.org. ^[1] Cure Sanfilippo Foundation, Columbia, SC, USA. ^[2] Patient and Policy Affairs, Lysogene, Neuilly sur Seine, France. ^[3] University of Pittsburgh Medical Center, Pittsburgh, PA, USA.;Children's Hospital of Pittsburgh of UPMC, Pittsburgh, PA, USA. ^[4] Cure Sanfilippo Foundation Parent Advocates, Columbia, SC, USA. ^[5] Center for Genomics, Bioinformatics, and Translational Research, RTI International, Research Triangle Park, NC, USA. ^[6]

关键词 MPS Meaningful treatment benefit Outcome measure Patient experience data Patient-focused drug development Sanfilippo syndrome

DOI 10.1007/s40120-020-00226-z

PMID 33263924

发布时间 2021-06-04

翻译满意度评价：

准确一般字义偏差语意偏离译文作用小提交

浏览0

Neurology and therapy

2021年10卷1期

197-212页

相似文献

中文期刊
外文期刊
学位论文
会议论文

浏览0

点击下载本期目录封面

Neurology and therapy

2021年10卷1期

197-212页

检索历史清除

Parent Experiences of Sanfilippo Syndrome Impact and Unmet Treatment Needs: A Qualitative Assessment.

Neurology and therapy

相似文献

Neurology and therapy

相关期刊

参考文献：
指定格式：	NoteExpress EndNote RefWorks NoteFirst

检索历史 清除

Parent Experiences of Sanfilippo Syndrome Impact and Unmet Treatment Needs: A Qualitative Assessment.

Neurology and therapy

相似文献

Neurology and therapy

相关期刊

检索历史清除