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Bone marrow transplantation for glycogen storage disease type II (Pompé's disease).

作者信息

Watson J G, Gardner-Medwin D, Goldfinch M E, Pearson A D

出版信息

N Engl J Med. 1986 Feb 6;314(6):385. doi: 10.1056/nejm198602063140611.

DOI:10.1056/nejm198602063140611
PMID:3511378
Abstract
摘要

相似文献

1
Bone marrow transplantation for glycogen storage disease type II (Pompé's disease).糖原贮积症II型(庞贝氏病)的骨髓移植
N Engl J Med. 1986 Feb 6;314(6):385. doi: 10.1056/nejm198602063140611.
2
More on bone marrow transplantation for glycogen storage disease type II (Pompé's disease).关于糖原贮积病II型(庞贝氏病)的骨髓移植更多信息。
N Engl J Med. 1986 Nov 6;315(19):1229.
3
Bone marrow transplantation for Pompé's disease.庞贝氏病的骨髓移植
N Engl J Med. 1986 Jul 3;315(1):65-6. doi: 10.1056/NEJM198607033150115.
4
Bone marrow transplantation in type IIa glycogen storage disease.IIa型糖原贮积病的骨髓移植
Birth Defects Orig Artic Ser. 1986;22(1):119-32.
5
Pompe's Disease Successfully Treated In Utero.
Am J Med Genet A. 2023 Mar;191(3):654-655. doi: 10.1002/ajmg.a.62795.
6
Natural bone marrow transplantation in cattle with Pompe's disease.患有庞贝氏病的牛的天然骨髓移植。
Neuromuscul Disord. 1991;1(6):449-54. doi: 10.1016/0960-8966(91)90008-g.
7
Anaesthesia for diagnostic muscle biopsy in an infant with Pompe's disease.庞贝病婴儿诊断性肌肉活检的麻醉
Can Anaesth Soc J. 1986 Nov;33(6):790-4. doi: 10.1007/BF03027132.
8
[Pompe's disease or glycogen storage disease].
Duodecim. 1982;98(9):709-16.
9
Echocardiographic evidence of outflow tract obstruction in Pompe's disease (glycogen storage disease of the heart).
Am J Cardiol. 1976 Jun;37(7):1103-6. doi: 10.1016/0002-9149(76)90433-1.
10
Unusual angiographic appearances of the left ventricle in 2 cases of Pompe's disease (glycogenosis type II).庞贝氏病(II型糖原贮积病)2例患者左心室异常血管造影表现。
Br Heart J. 1979 Feb;41(2):238-40. doi: 10.1136/hrt.41.2.238.

引用本文的文献

1
Advances in Pompe Disease Treatment: From Enzyme Replacement to Gene Therapy.庞贝病治疗进展:从酶替代疗法到基因治疗。
Mol Diagn Ther. 2024 Nov;28(6):703-719. doi: 10.1007/s40291-024-00733-x. Epub 2024 Aug 12.
2
Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases.基因治疗对溶酶体贮积症心血管症状的影响。
Genet Mol Biol. 2019;42(1 suppl 1):261-285. doi: 10.1590/1678-4685-GMB-2018-0100. Epub 2019 May 23.
3
Pompe disease diagnosis and management guideline.庞贝病诊断与管理指南。
Genet Med. 2006 May;8(5):267-88. doi: 10.1097/01.gim.0000218152.87434.f3.
4
Clinical and metabolic correction of pompe disease by enzyme therapy in acid maltase-deficient quail.酸性麦芽糖酶缺乏鹌鹑中酶疗法对庞贝病的临床和代谢纠正
J Clin Invest. 1998 Feb 15;101(4):827-33. doi: 10.1172/JCI1722.
5
Prospect for enzyme therapy in glycogenosis II variants: a study on cultured muscle cells.
J Neurol. 1988 Sep;235(7):392-6. doi: 10.1007/BF00314479.
6
Muscle glycogenosis.
J Inherit Metab Dis. 1990;13(4):452-65. doi: 10.1007/BF01799503.
7
Intravenous administration of phosphorylated acid alpha-glucosidase leads to uptake of enzyme in heart and skeletal muscle of mice.静脉注射磷酸化酸性α-葡萄糖苷酶会导致该酶在小鼠的心脏和骨骼肌中被摄取。
J Clin Invest. 1991 Feb;87(2):513-8. doi: 10.1172/JCI115025.