View from inside: Rare diseases in the times of COVID19.
作者信息
Vockley Jerry
机构信息
University of Pittsburgh School of Medicine and Graduate School of Public Health, UPMC Children's Hospital of Pittsburgh, Center for Rare Disease Therapy, Pittsburgh, Pennsylvania, USA.
出版信息
J Inherit Metab Dis. 2021 Jan;44(1):2-3. doi: 10.1002/jimd.12334. Epub 2020 Dec 2.
Abstract
摘要
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J Inherit Metab Dis. 2019 Jan;42(1):169-177. doi: 10.1002/jimd.12038.
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Triheptanoin versus trioctanoin for long-chain fatty acid oxidation disorders: a double blinded, randomized controlled trial.三庚酸甘油酯与三辛酸甘油酯治疗长链脂肪酸氧化障碍：一项双盲、随机对照试验。
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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.长链脂肪酸氧化障碍患者在转换为三庚酸甘油酯治疗前后的长期主要临床结局——一项回顾性病历审查
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