第一单位：
Washington University School of Medicine, St Louis, MO, USA.
作者：
Daniel S,Ory [1]
;
Elizabeth A,Ottinger [2]
;
Nicole Yanjanin,Farhat [3]
;
Kelly A,King [4]
;
Xuntian,Jiang [1]
;
Lisa,Weissfeld [5]
;
Elizabeth,Berry-Kravis [6]
;
Cristin D,Davidson [7]
;
Simona,Bianconi [3]
;
Lee Ann,Keener [3]
;
Ravichandran,Rao [8]
;
Ariane,Soldatos [9]
;
Rohini,Sidhu [1]
;
Kimberly A,Walters [5]
;
Xin,Xu [2]
;
Audrey,Thurm [10]
;
Beth,Solomon [11]
;
William J,Pavan [12]
;
Bernardus N,Machielse [8]
;
Mark,Kao [13]
;
Steven A,Silber [14]
;
John C,McKew [2]
;
Carmen C,Brewer [4]
;
Charles H,Vite [15]
;
Steven U,Walkley [7]
;
Christopher P,Austin [2]
;
Forbes D,Porter [16]
作者单位：
Washington University School of Medicine, St Louis, MO, USA.
[1]
National Center for Advancing Translational Sciences, National Institutes of Health, Department of Health and Human Services, Bethesda, MD, USA.
[2]
Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Department of Health and Human Services, Bethesda, MD USA.
[3]
National Institute on Deafness and Other Communication Disorders, National Institutes of Health, Department of Health and Human Services, Bethesda, MD, USA.
[4]
Statistics Collaborative, Washington, DC, USA.
[5]
Rush University Medical Center, Chicago, IL, USA.
[6]
Albert Einstein College of Medicine, Bronx, NY, USA.
[7]
Vtesse Inc, Gaithersburg, MD, USA.
[8]
National Institute of Neurological Disorders and Stroke, National Institutes of Health, Department of Health and Human Services Bethesda, MD, USA.
[9]
National Institute of Mental Health, National Institutes of Health, Department of Health and Human Services, Bethesda, MD, USA.
[10]
Mark O Hatfield Clinical Research Center, National Institutes of Health, Department of Health and Human Services, Bethesda, MD, USA.
[11]
National Human Genome Research Institute, National Institutes of Health, Department of Health and Human Services, Bethesda, MD, USA.
[12]
Preclinical Development and Safety, Janssen R&D, Raritan, NJ, USA.
[13]
Global Public Health, Johnson & Johnson, Philadelphia, PA, USA.
[14]
School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA, USA.
[15]
Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Department of Health and Human Services, Bethesda, MD USA. Electronic address: fdporter@mail.nih.gov.
[16]
医学主题词
青少年(Adolescent);儿童(Child);儿童, 学龄前(Child, Preschool);疾病恶化(Disease Progression);剂量效应关系, 药物(Dose-Response Relationship, Drug);女(雌)性(Female);听觉丧失, 高频(Hearing Loss, High-Frequency);人类(Humans);羟基胆甾醇类(Hydroxycholesterols);注射, 脊髓(Injections, Spinal);男(雄)性(Male);尼曼-皮克病, C型(Niemann-Pick Disease, Type C);少见病(Rare Diseases);青年人(Young Adult)
DOI
10.1016/S0140-6736(17)31465-4
PMID
28803710
发布时间
2025-05-30
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