Long-term safety and efficacy of DTX301 in adults with late-onset ornithine transcarbamylase (OTC) deficiency: A Phase 1/2 trial

被引：1

作者：

Thomas, Janet ^{[1
,2
]}

Tan, Wen-Hann ^{[3
]}

Khan, Aneal ^{[4
,5
]}

Konczal, Laura ^{[6
]}

Breilyn, Margo Sheck ^{[7
]}

Hualde, Leticia Ceberio ^{[8
]}

Couce, Marie Luz ^{[9
]}

Geberhiwot, Tarekegn ^{[10
]}

Guffon, Nathalie ^{[11
]}

Harding, Cary ^{[12
]}

Patra, Kaushik ^{[13
]}

Merritt, J. Lawrence, II ^{[13
]}

Crombez, Eric ^{[13
]}

机构：

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

[2] Childrens Hosp Colorado, Aurora, CO USA

[3] Harvard Med Sch, Boston Childrens Hosp, Boston, MA 02115 USA

[4] MAGIC Clin Ltd, Calgary, AB, Canada

[5] Univ Calgary, Calgary, AB, Canada

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

[7] Icahn Sch Med Mt Sinai, New York, NY 10029 USA

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

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

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

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

[12] Oregon Hlth & Sci Univ, Portland, OR 97201 USA

[13] Ultragenyx Pharmaceut Inc, Novato, CA USA

来源：

MOLECULAR GENETICS AND METABOLISM | 2024年 / 141卷 / 04期

关键词：

D O I：

10.1016/j.ymgme.2024.108266

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Poster # 0

引用

页数：2

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