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Elivaldogene autotemcel approved for treatment of cerebral adrenoleukodystrophy (CALD) in males: A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG).

作者信息

Scott Anna I, Mallhi Kanwaldeep K, Ganesh Jaya, Chen Wei-Liang

机构信息

Department of Laboratories, Seattle Children's Hospital, Seattle, WA.

Department of Laboratory Medicine and Pathology, University of Washington, Seattle, WA.

出版信息

Genet Med Open. 2023 Nov 15;1(1):100835. doi: 10.1016/j.gimo.2023.100835. eCollection 2023.

DOI:10.1016/j.gimo.2023.100835
PMID:39669257
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11613678/
Abstract
摘要

相似文献

1
Elivaldogene autotemcel approved for treatment of cerebral adrenoleukodystrophy (CALD) in males: A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG).艾利伐多基因自体T细胞疗法获批准用于治疗男性脑肾上腺白质营养不良(CALD):美国医学遗传学与基因组学学会(ACMG)的治疗公告
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2
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Casgevy (exagamglogene autotemcel) and Lyfgenia (lovotibeglogene autotemcel) for individuals 12 years and older with sickle cell disease (SCD) and recurrent vaso-occlusive crises (VOC): A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG).适用于12岁及以上患有镰状细胞病(SCD)且反复发生血管闭塞性危机(VOC)的个体的Casgevy(exagamglogene autotemcel)和Lyfgenia(lovotibeglogene autotemcel):美国医学遗传学与基因组学学会(ACMG)的治疗公告
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Genet Med Open. 2023 Mar 27;1(1):100771. doi: 10.1016/j.gimo.2023.100771. eCollection 2023.
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Evaluation of Neurofilament Light Chain as a Biomarker of Neurodegeneration in X-Linked Childhood Cerebral Adrenoleukodystrophy.评估神经丝轻链作为 X 连锁儿童脑肾上腺脑白质营养不良神经退行性变的生物标志物。
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本文引用的文献

1
Disease progression in women with X-linked adrenoleukodystrophy is slow.女性 X 连锁肾上腺脑白质营养不良的疾病进展缓慢。
Orphanet J Rare Dis. 2019 Feb 7;14(1):30. doi: 10.1186/s13023-019-1008-6.
2
Adrenoleukodystrophy: Guidance for Adrenal Surveillance in Males Identified by Newborn Screen.肾上腺脑白质营养不良:新生儿筛查发现的男性患者的肾上腺监测指南。
J Clin Endocrinol Metab. 2018 Nov 1;103(11):4324-4331. doi: 10.1210/jc.2018-00920.
3
Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.用于脑型肾上腺脑白质营养不良的造血干细胞基因治疗
N Engl J Med. 2017 Oct 26;377(17):1630-1638. doi: 10.1056/NEJMoa1700554. Epub 2017 Oct 4.
4
Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy.采用慢病毒载体的造血干细胞基因疗法治疗X连锁肾上腺脑白质营养不良症
Science. 2009 Nov 6;326(5954):818-23. doi: 10.1126/science.1171242.