PLASMA INFUSION THERAPY FOR HURLER'S SYNDROME

[摘要] 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.

[学科分类] 儿科学