Long-Term Safety and Urea Cycle Function Following a Phase 1/2 Trial of DTX301 in Adults with Late-Onset Ornithine Transcarbamylase (OTC) Deficiency

被引：0

作者：

Harding, Cary O. ^{[1
]}

Geberhiwot, Tarekegn ^{[2
]}

Couce, Maria Luz ^{[3
]}

Guffon, Nathalie ^{[4
]}

Tan, Wen-Hann ^{[5
]}

Hualde, Leticia Ceberio ^{[6
]}

Khan, Aneal ^{[7
]}

Konczal, Laura ^{[8
]}

Thomas, Janet A. ^{[9
,10
]}

Breilyn, Margo Sheck ^{[11
]}

Zhang, Lin ^{[12
]}

Merritt, J. Lawrence ^{[12
]}

Crombez, Eric ^{[12
]}

机构：

[1] Oregon Hlth & Sci Univ, Portland, OR USA

[2] Univ Birmingham, Birmingham, W Midlands, England

[3] Univ Santiago Compostela, Santiago De Compostela, Spain

[4] HCL, Reference Ctr Inherited Metab Dis, Lyon, France

[5] Boston Childrens Hosp, Boston, MA USA

[6] Cruces Univ Hosp, Biocruces Bizkaia Hlth Res Inst, Baracaldo, Spain

[7] MAGic Clin Ltd, Calgary, AB, Canada

[8] Univ Hosp Cleveland Med Ctr, Cleveland, OH USA

[9] Univ CO, Sch Med, Aurora, CO USA

[10] Childrens Hosp Colorado, Aurora, CO USA

[11] Icahn Sch Med Mt Sinai, New York, NY USA

[12] Ultragenyx Pharmaceut Inc, Novato, CA USA

来源：

关键词：

D O I：

暂无

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

1411

引用

页码：668 / 669

页数：2

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