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

页码：S349 / S349

页数：1

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