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
相关论文
共 50 条
  • [41] Long-term safety and efficacy of zafirlukast in the treatment of asthma: interim results of an open-label extension trial
    Grossman, J
    Smith, LJ
    Wilson, AM
    Thyrum, PT
    ANNALS OF ALLERGY ASTHMA & IMMUNOLOGY, 1999, 82 (04) : 361 - 369
  • [42] Early results from a long-term disease monitoring program in patients with long-chain fatty acid oxidation disorders
    Sanchez-Valle, Amarilis
    Vockley, Jerry
    Burton, Barbara
    Inbar-Feigenberg, Michal
    Aggarwal, Anjali
    Hetzer, Joel
    Reineking, Bridget
    Grimm, Andrew
    MOLECULAR GENETICS AND METABOLISM, 2024, 141 (04)
  • [43] Safety of long-term use of linezolid: results of an open-label study
    Vazquez, Jose A.
    Arnold, Anthony C.
    Swanson, Robert N.
    Biswas, Pinaki
    Bassetti, Matteo
    THERAPEUTICS AND CLINICAL RISK MANAGEMENT, 2016, 12 : 1347 - 1354
  • [44] Safety and efficacy of tofacitinib for up to 9.5years in the treatment of rheumatoid arthritis: final results of a global, open-label, long-term extension study
    Wollenhaupt, Juergen
    Lee, Eun-Bong
    Curtis, Jeffrey R.
    Silverfield, Joel
    Terry, Ketti
    Soma, Koshika
    Mojcik, Chris
    DeMasi, Ryan
    Strengholt, Sander
    Kwok, Kenneth
    Lazariciu, Irina
    Wang, Lisy
    Cohen, Stanley
    ARTHRITIS RESEARCH & THERAPY, 2019, 21 (1)
  • [45] LONG-TERM TREATMENT OF PARTIAL EPILEPSY WITH ESLICARBAZEPINE ACETATE: RESULTS OF A 1-YEAR OPEN-LABEL EXTENSION STUDY
    Guekht, A.
    Elger, C.
    Halasz, P.
    Maia, J.
    Almeida, L.
    Soares-da-Silva, P.
    EPILEPSIA, 2009, 50 : 106 - 106
  • [46] LONG-TERM SAFETY AND EFFICACY OF RISANKIZUMAB TREATMENT IN PATIENTS WITH CROHN'S DISEASE: FINAL RESULTS FROM THE PHASE 2 OPEN-LABEL EXTENSION STUDY
    Ferrante, Marc
    Feagan, Brian G.
    Panes, Julian
    Baert, Filip J.
    Louis, Edouard
    Dewit, Olivier
    Kaser, Arthur
    Duan, Weijiang
    Gustafson, Dawn
    Liao, Xiaomei
    Wallace, Kori
    Kalabic, Jasmina
    D'Haens, Geert R.
    GASTROENTEROLOGY, 2020, 158 (06) : S1198 - S1198
  • [47] Long-term safety and efficacy of risankizumab treatment in patients with Crohn's disease: Final results from the Phase 2 open-label extension study
    Ferrante, M.
    Feagan, B. G.
    Panes, J.
    Baert, F.
    Louis, E.
    Dewit, O.
    Kaser, A.
    Duan, W. R.
    Gustafson, D.
    Liao, X.
    Wallace, K.
    Kalabic, J.
    D'Haens, G. R.
    JOURNAL OF CROHNS & COLITIS, 2020, 14 : S024 - S025
  • [48] Effect of brexpiprazole on long-term remission in adults with schizophrenia: results of an open-label, long-term study
    Baker, R.
    Jin, N.
    Weiller, E.
    Weiss, C.
    EUROPEAN NEUROPSYCHOPHARMACOLOGY, 2017, 27 : S952 - S952
  • [49] Long-Term Treatment With Deutetrabenazine Is Associated With Continued Improvement in Tardive Dyskinesia: Results From an Open-Label Extension Study
    Hauser, R.
    Barkay, H.
    Fernandez, H.
    Factor, S.
    Jimenez-Shahed, J.
    Gross, N.
    Gordon, M.
    Savola, J. M.
    Anderson, K.
    MOVEMENT DISORDERS, 2019, 34 : S596 - S596
  • [50] Ketone supplementation before exercise in long-chain fatty acid oxidation disorder (LC-FAOD) patients suppressed lipolysis despite mild increases in blood ketones
    Gregor, A. N.
    Delerive, P.
    Cuenoud, B.
    Harding, C. O.
    Gillingham, M. B.
    MOLECULAR GENETICS AND METABOLISM, 2024, 141 : 30 - 31
12345