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頁籤選單縮合
題名 | 龐貝氏症之治療=Current Therapy of Pompe Disease |
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作者 | 黃建衡; 施嘉慶; 李威; 謝靜婷; 王茂富; | 書刊名 | 藥學雜誌 |
卷期 | 27:1=106 2011.03[民100.03] |
頁次 | 頁61-64 |
分類號 | 417.509 |
關鍵詞 | 龐貝氏症; α-葡萄糖苷酶; 酵素替代療法; Pompe disease; GAA; ERT; |
語文 | 中文(Chinese) |
中文摘要 | 龐貝氏症 (肝醣儲積症 )是一種代謝性肌肉疾病,其發生的主要原因是缺乏了可於溶小體內分解多餘肝醣之α -葡萄糖苷酶。該病患者通常會有肌肉衰弱、呼吸無力的症狀且情況會越來越嚴重,尤其在嬰兒患者中往往可以發現心臟肥大的情況,隨著這些問題的日益惡化亦很有可能造成日後心肺衰竭甚至死亡的危險因子。目前由國人研發成功的酵素替代療法已經被美國食品藥物管理局 FDA於2006及2010年核准用來治療嬰兒型與晚發型的龐貝氏症。此療法是利用基因工程的技術所製成的外生性酵素,將病人所缺乏的 GAA酵素定期注射至血液中,來幫助溶小體內肝醣的分解,改善肌肉、呼吸功能提升患者的存活率,目前健保可依罕見疾病法給付。 |
英文摘要 | Pompe disease (glycogen storage disease II) is a metabolic myopathy caused by a deficiency of acid α glucosidase (GAA), an enzyme that degrades lysosomal glycogen. The devastating disease occurs in an estimated 1 in every 40,000 to 300,000 births. Its primary symptom is heart and skeletal muscle weakness, progressing to respiratory weakness and death from respiratory failure. The efficacy of enzyme replacement therapy (ERT) with recombinant human GAA was demonstrated during clinical trials that prolonged subjects overall survival, prolonged ventilator-free survival, and also improved cardiomyopathy, which led to broad-label approval by the U. S. Food and Drug Administration. |
本系統之摘要資訊系依該期刊論文摘要之資訊為主。