Lenmeldy (atidarsagene autotemcel) for individuals with early metachromatic leukodystrophy (MLD): A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG).
作者信息
Gangji Rahaman Navaz, Ali Qais Abu, Dhamija Radhika, Corado Andrés Morales, Kharbanda Sandhya
机构信息
Division of Medical Genetics, Department of Pediatrics, University of California San Francisco, San Francisco, CA.
qRare International, LLC, Pleasanton, CA.
出版信息
Genet Med Open. 2025 Jun 24;3:103432. doi: 10.1016/j.gimo.2025.103432. eCollection 2025.
Abstract
摘要
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Lenmeldy (atidarsagene autotemcel) for individuals with early metachromatic leukodystrophy (MLD): A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG).用于早发性异染性脑白质营养不良（MLD）患者的Lenmeldy（阿替达萨基因自体T细胞）：美国医学遗传学与基因组学学会（ACMG）的治疗公告
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本文引用的文献
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Developmental delay can precede neurologic regression in early onset metachromatic leukodystrophy.在早发型异染性脑白质营养不良中，发育迟缓可能先于神经功能衰退出现。
Mol Genet Metab. 2024 Aug;142(4):108521. doi: 10.1016/j.ymgme.2024.108521. Epub 2024 Jun 29.
2
Atidarsagene autotemcel for metachromatic leukodystrophy.用于治疗异染性脑白质营养不良的阿替达萨基因自体T细胞疗法。
J Manag Care Spec Pharm. 2024 Feb 3;30(2):201-205. doi: 10.18553/jmcp.2024.30.2.201.
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Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access.慢病毒造血干细胞基因治疗早发性异染性脑白质营养不良：1/2 期非随机、开放标签、单臂临床试验及扩大使用的长期结果。
Lancet. 2022 Jan 22;399(10322):372-383. doi: 10.1016/S0140-6736(21)02017-1.
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Metachromatic leukodystrophy: A single-center longitudinal study of 45 patients.脑硫脂沉积病：45 例患者的单中心纵向研究。
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Science. 2013 Aug 23;341(6148):1233158. doi: 10.1126/science.1233158. Epub 2013 Jul 11.
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