IMPROVEMENTS IN MAJOR CLINICAL EVENTS IN CHILDREN TREATED WITH TRIHEPTANOIN FOR LONG-CHAIN FATTY ACID OXIDATION DISORDERS (LC-FAOD)

被引：0
|
作者：
Ray, Kathryn [1 ]
Pisani, Laura [1 ]
Lu, Xiaoxiao [1 ]
Vockley, Jerry [2 ]
机构：
[1] Ultragenyx Pharmaceut Inc, Novato, CA USA
[2] Univ Pittsburgh, Sch Med, UPMC Childrens Hosp Pittsburgh, Pittsburgh, PA USA
来源：
MOLECULAR GENETICS AND METABOLISM | 2022年 / 135卷 / 04期
关键词：
D O I：
暂无
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
75
引用
收藏
页码：295 / 296
页数：2
相关论文
共 50 条
  • [41] Resting and total energy expenditure of patients with long-chain fatty acid oxidation disorders (LC-FAODs)
    DeLany, James P.
    Horgan, Angela
    Gregor, Ashley
    Vockley, Jerry
    Harding, Cary O.
    Gillingham, Melanie B.
    MOLECULAR GENETICS AND METABOLISM, 2023, 138 (03)
  • [42] Resting and total energy expenditure of patients with long-chain fatty acid oxidation disorders (LC-FAODs)
    Gillingham, M. G.
    Gregor, A.
    Vockley, J.
    Harding, C. O.
    DeLany, J. P.
    MOLECULAR GENETICS AND METABOLISM, 2022, 136 : S14 - S14
  • [43] RESULTS FROM A 78-WEEK SINGLE-ARM, OPEN-LABEL PHASE 2 STUDY TO EVALUATE UX007 IN PEDIATRIC AND ADULT PATIENTS WITH MODERATE TO SEVERE LONG-CHAIN FATTY ACID OXIDATION DISORDERS (LC-FAOD)
    Vockley, Jerry
    Burton, Barbara
    Berry, Gerard T.
    Longo, Nicola
    Phillips, John
    Sanchez-Valle, Amarilis
    Tanpaiboon, Pranoot
    Grunewald, Stephanie
    Murphy, Elaine
    Bowden, Alexandra
    Chen, Wencong
    Mu, Yunming
    Cataldo, Jason
    Marsden, Deborah
    Kakkis, Emil
    MOLECULAR GENETICS AND METABOLISM, 2018, 123 (03) : 274 - 275
  • [44] Long-term major clinical outcomes in patients with long chain fatty acid oxidation disorders before and after transition to triheptanoin treatment-A retrospective chart review
    Vockley, Jerry
    Marsden, Deborah
    McCracken, Elizabeth
    DeWard, Stephanie
    Barone, Amanda
    Hsu, Kristen
    Kakkis, Emil
    MOLECULAR GENETICS AND METABOLISM, 2015, 116 (1-2) : 53 - 60
  • [45] Different Lipid Signature in Fibroblasts of Long-Chain Fatty Acid Oxidation Disorders
    Alatibi, Khaled I.
    Hagenbuchner, Judith
    Wehbe, Zeinab
    Karall, Daniela
    Ausserlechner, Michael J.
    Vockley, Jerry
    Spiekerkoetter, Ute
    Gruenert, Sarah C.
    Tucci, Sara
    CELLS, 2021, 10 (05)
  • [46] Clinical outcomes in a series of 18 patients with long chain fatty acids oxidation disorders treated with triheptanoin for a median duration of 22 months
    Guffon, Nathalie
    Mochel, Fanny
    Schiff, Manuel
    De Lonlay, Pascale
    Douillard, Claire
    Vianey-Saban, Christine
    MOLECULAR GENETICS AND METABOLISM, 2021, 132 (04) : 227 - 233
  • [47] UNDERSTANDING THE BURDEN OF LONG-CHAIN FATTY ACID OXIDATION DISORDERS FOR PATIENTS AND CAREGIVERS
    Kruger, E.
    Voorhees, K.
    Thomas, N. A.
    Judge, M.
    Galla, J.
    Kung, J.
    Rodriguez, D.
    VALUE IN HEALTH, 2021, 24 : S205 - S205
  • [48] Understanding the impact of long-chain fatty acid oxidation disorders for patients and caregivers
    Kruger, Eliza
    Voorhees, Kristin
    Thomas, Nina
    Judge, Miller
    Galla, John
    Kung, Jeffrey
    Rodriguez, Diego
    MOLECULAR GENETICS AND METABOLISM REPORTS, 2022, 32
  • [49] QUALITATIVE EXPLORATION OF THE PATIENT EXPERIENCE OF LONG-CHAIN FATTY ACID OXIDATION DISORDERS
    Hall, R.
    Kruger, E.
    Bowden, A.
    Cimms, T.
    Skrinar, A.
    Tinsley, K.
    Gater, A.
    VALUE IN HEALTH, 2020, 23 : S342 - S343
  • [50] Concerning "Triheptanoin vs trioctanoin for long-chain fatty acid oxidation disorders: A double blinded, randomized controlled trial" by Gillingham et al.
    Shoffner, John M.
    JOURNAL OF INHERITED METABOLIC DISEASE, 2019, 42 (03) : 394 - 395
12345