Outcomes of Long-Term Treatment with Laronidase in Patients with Mucopolysaccharidosis Type I
Author:
Funder
Sanofi Genzyme
Publisher
Elsevier BV
Subject
Pediatrics, Perinatology and Child Health
Reference30 articles.
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2. Mucopolysaccharidosis I: management and treatment guidelines;Muenzer;Pediatrics,2009
3. The natural history of MPS I: global perspectives from the MPS I Registry;Beck;Genet Med,2014
4. Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase);Wraith;J Pediatr,2004
5. Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler's syndrome after pharmacological enzyme replacement therapy;Saif;Haematologica,2012
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