Ontology highlight
ABSTRACT:
SUBMITTER: Dickson PI
PROVIDER: S-EPMC4572891 | biostudies-literature | 2015 Sep-Oct
REPOSITORIES: biostudies-literature
Dickson Patricia I PI Kaitila Ilkka I Harmatz Paul P Mlikotic Anton A Chen Agnes H AH Victoroff Alla A Passage Merry B MB Madden Jacqueline J Le Steven Q SQ Naylor David E DE
Molecular genetics and metabolism 20150726 1-2
Enzyme replacement therapy with laronidase (recombinant human alpha-l-iduronidase) is successfully used to treat patients with mucopolysaccharidosis type I (MPS I). However, the intravenously-administered enzyme is not expected to treat or prevent neurological deterioration. As MPS I patients suffer from spinal cord compression due in part to thickened spinal meninges, we undertook a phase I clinical trial of lumbar intrathecal laronidase in MPS I subjects age 8 years and older with symptomatic ...[more]