Abstract

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.

Full Text
Paper version not known

Talk to us

Join us for a 30 min session where you can share your feedback and ask us any queries you have

Schedule a call

Disclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.