%0 Journal Article
%T Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series
%A Ana Carolina Esposito
%A Ana Maria Martins
%A Angelina X. Acosta
%A Anna Hlavat¨¢
%A Anneliese Lopes Barth
%A Carolina Fischinger Moura De Souza
%A Dafne Dain Gandelman Horovitz
%A Em¨ªlia Katiane Embiru£¿u de Ara¨²jo Le£¿o
%A Katar¨ªna Hlavat¨¢
%A Laercio Cardoso
%A Jr.
%A Michel C. Tchan
%A Roberto Giugliani
%A Sandra Obikawa Kyosen
%J Archive of "Orphanet Journal of Rare Diseases".
%D 2016
%R 10.1186/s13023-016-0437-8
%X Enzyme replacement therapy (ERT) with laronidase (recombinant human ¦Á-L-iduronidase, Aldurazyme£¿) is indicated for non-neurological signs and symptoms of mucopolysaccharidosis type I (MPS I). The approved laronidase dose regimen is weekly infusions of 0.58mg/kg, however, patients and caregivers may have difficulty complying with the weekly regimen. We examined clinical outcomes, tolerability, compliance, and satisfaction in a series of patients who switched to every other week infusions
%K Enzyme replacement therapy
%K ¦Á-L-iduronidase deficiency
%K Clinical outcomes
%K Tolerability
%U https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4850670/