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
收藏
页数：2
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