Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retr...
Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series
About this item
Full title
Author / Creator
Horovitz, Dafne Dain Gandelman , Acosta, Angelina X , Giugliani, Roberto , Hlavatá, Anna , Hlavatá, Katarína , Tchan, Michel C , Lopes Barth, Anneliese , Cardoso, Jr, Laercio , Embiruçu de Araújo Leão, Emília Katiane , Esposito, Ana Carolina , Kyosen, Sandra Obikawa , De Souza, Carolina Fischinger Moura and Martins, Ana Maria
Publisher
England: BioMed Central Ltd
Journal title
Language
English
Formats
Publication information
Publisher
England: BioMed Central Ltd
Subjects
More information
Scope and Contents
Contents
Enzyme replacement therapy (ERT) with laronidase (recombinant human α-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....
Alternative Titles
Full title
Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series
Authors, Artists and Contributors
Author / Creator
Acosta, Angelina X
Giugliani, Roberto
Hlavatá, Anna
Hlavatá, Katarína
Tchan, Michel C
Lopes Barth, Anneliese
Cardoso, Jr, Laercio
Embiruçu de Araújo Leão, Emília Katiane
Esposito, Ana Carolina
Kyosen, Sandra Obikawa
De Souza, Carolina Fischinger Moura
Martins, Ana Maria
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4850670
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4850670
Other Identifiers
ISSN
1750-1172
E-ISSN
1750-1172
DOI
10.1186/s13023-016-0437-8
