Triheptanoin for the treatment of Long-Chain Fatty Acid Disorders (LC-FAOD): Final results of an open-label, long-term extension study

被引：2
|
作者：
Vockley, Jerry [1 ]
Burton, Barbara [2 ]
Berry, Gerard [3 ]
Longo, Nicola [4 ]
Phillips, John [5 ]
Sanchez-Valle, Amarilis [6 ]
Chapman, Kimberly [7 ]
Tanpaiboon, Pranoot [7 ]
Grunewald, Stephanie [8 ,9 ]
Murphy, Elaine [10 ]
Lu, Xiaoxiao [11 ]
Rahman, Syeda [11 ]
Ray, Kathryn [11 ]
Ramirez, Antonio Nino [11 ]
机构：
[1] Univ Pittsburgh, Pittsburgh, PA USA
[2] Ann & Robert H Lurie Childrens Hosp, Chicago, IL USA
[3] Boston Children Hosp, Boston, MA USA
[4] Univ Utah, Salt Lake City, UT 84112 USA
[5] Vanderbilt Univ, Med Ctr, Nashville, TN USA
[6] USF Hlth, Morsani Coll Med, Tampa, FL USA
[7] Childrens Natl Hlth Syst, Washington, DC USA
[8] Great Ormond St Hosp Sick Children, London, England
[9] UCL Inst Child Hlth, NIHR BRC, London, England
[10] Natl Hosp Neurol & Neurosurg, London, England
[11] Ultragenyx Pharmaceut Inc, Novato, CA USA
来源：
GENETICS IN MEDICINE | 2022年 / 24卷 / 03期
关键词：
D O I：
10.1016/j.gim.2022.01.567
中图分类号：
Q3 [遗传学];
学科分类号：
071007 ; 090102 ;
摘要：
OP017
引用
收藏
页码：S349 / S349
页数：1
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