Enzyme replacement therapy started at birth improves outcome in difficult-to-treat organs in mucopolysaccharidosis I mice-Reference-Cited by-同舟云学术

Enzyme replacement therapy started at birth improves outcome in difficult-to-treat organs in mucopolysaccharidosis I mice

Published:2013-05 Issue:1 Volume:109 Page:33-40
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Baldo Guilherme,Mayer Fabiana Q.,Martinelli Bárbara Z.,de Carvalho Talita G.,Meyer Fabiola S.,de Oliveira Patrícia G.,Meurer Luise,Tavares Ângela,Matte Ursula,Giugliani Roberto

Funder

Conselho Nacional de Desenvolvimento Cientifico (CNPq)

FAPERGS

Fundo de Incentivo a Pesquisa do HCPA (FIPE-HCPA)

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference31 articles.

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2. Mucopolysaccharidosis I, II, and VI: brief review and guidelines for treatment;Giugliani;Genet. Mol. Biol.,2010

3. Cell microencapsulation: a potential tool for the treatment of neuronopathic lysosomal storage diseases;Matte;J. Inherit. Metab. Dis.,2011

4. Replacing the enzyme alpha-l-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I;Dierenfeld;Sci. Transl. Med.,2010

5. Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human alpha-l-iduronidase (laronidase);Wraith;Pediatrics,2007

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