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PLASMA INFUSION THERAPY FOR HURLER'S SYNDROME

机译:Hurler综合征的等离子输注治疗

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Ever since the first report from Houston of the "plasma infusion" approach to modification of the Hurler's syndrome picture there has been a wide popular feeling that the beginnings of a "treatment" were at hand for this group of inborn error diseases. Intriguing data on alteration of the pattern of urinary mucopolysaccharide excretion, and, to some extent, of the children's clinical manifestations, opened a real hope that a substantial mechanism existed for rendering more normal the skewed metabolism of these patients. In addition, the authors commented that "human plasma infusions . . . seemed to fit the recommendations" given in a previous PEDIATRICS Commentary for theoretically useful therapeutic trials of new materials in patients with inborn error syndromes.
机译:自从休斯敦首次发表关于“血浆输注”方法改善霍勒氏综合症图片的报道以来,人们普遍认为,“治疗”的开始就在这一组先天性错误疾病的治疗上。关于尿液黏多糖分泌模式改变以及在某种程度上儿童临床表现变化的有趣数据,真正希望存在一种实质性的机制,以使这些患者的新陈代谢更加正常。另外,作者评论说,先前的《 PEDIATRICS评论》中给出的“人血浆输注……似乎符合建议”,用于在先天性错误综合征患者中对新材料进行理论上有用的治疗性试验。

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