ENZYME REPLACEMENT THERAPY IN TREATMENT OF MUCOPOLYSACCHARIDOSIS I

$982M01FY2000RRNIH

La Biomed Res Inst/ Harbor Ucla Med Ctr, Torrance CA

Investigators

Linked publications & trials

Paper 39397778 Paper 38880394 Paper 38746373 Paper 38547955 Paper 38236978 Paper 38222977 Paper 38190104 Paper 38160309 Paper 37910437 Paper 37777948 Paper 37662185 Paper 37659258 Paper 37338666 Paper 36780383 Paper 36540980 Paper 36128629 Paper 35472396 Paper 35244151 Paper 35175842 Paper 35113658 Paper 34966880 Paper 34752621 Paper 34728061 Paper 34570768 Paper 34528718 Paper 34460319 Paper 34166655 Paper 34161253 Paper 34038937 Paper 33901188 Paper 33662486 Paper 33545261 Paper 33270715 Paper 33202262 Paper 33066718 Paper 32984497 Paper 32936208 Paper 32928740 Paper 32803094 Paper 32578481 Paper 32556630 Paper 32492107 Paper 32458454 Paper 32375556 Paper 32268814 Paper 32226820 Paper 32063125 Paper 32063062 Paper 32037132 Paper 31957961 Paper 31940010 Paper 31884245 Paper 31859580 Paper 31772544 Paper 31714576 Paper 31564163 Paper 31547912 Paper 31490202 Paper 31448444 Paper 31404422 Paper 31058466 Paper 31056078 Paper 30976755 Paper 30888919 Paper 30801985 Paper 30661700 Paper 30496407 Paper 30487263 Paper 30470724 Paper 30370022 Paper 30341055 Paper 30336817 Paper 30293664 Paper 30214855 Paper 30041793 Paper 29985202 Paper 29920702 Paper 29762147 Paper 29738414 Paper 29670744 Paper 29575061 Paper 29459474 Paper 29263019 Paper 29217675 Paper 29150290 Paper 29103858 Paper 29081724 Paper 28938457 Paper 28931566 Paper 28607128 Paper 28605043 Paper 28428055 Paper 28158423 Paper 28109970 Paper 28081214 Paper 27618452 Paper 27507809 Paper 27438329 Paper 27267867 Paper 27190099

Abstract

This clinical study will evaluate the safety and efficacy ofenzyme replacement therapy in the treatment of MPS I, a lysosomal storage disorder. Children 5-18 years of age will be admitted, evaluated and infused with recombinant diuronidase. We will study the effects of the enzyme on lysosmal storage in the liver, spleen, urine and other sites as well as its safety.

View original record on NIH RePORTER →