Enzyme replacement therapy started at birth improves outcome in difficult-to-treat organs in mucopolysaccharidosis I mice (Q44516087)

3 December 2017

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

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enzyme replacement therapy

3 December 2017

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mucopolysaccharidosis I

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10

Roberto Giugliani

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3 December 2017

9

Ursula Matte

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3 December 2017

1

Guilherme Baldo

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3 December 2017

Fabiana Q Mayer

2

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3 December 2017

Bárbara Z Martinelli

3

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3 December 2017

Talita G de Carvalho

4

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3 December 2017

Fabiola S Meyer

5

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3 December 2017

Patrícia G de Oliveira

6

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3 December 2017

Luise Meurer

7

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3 December 2017

Angela Tavares

8

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3 December 2017

16 March 2013

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Molecular Genetics and Metabolism

3 December 2017

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3 December 2017

109

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3 December 2017

1

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3 December 2017

33-40

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Mucopolysaccharidosis I, II, and VI: Brief review and guidelines for treatment.

3 December 2017

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https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Cell microencapsulation: a potential tool for the treatment of neuronopathic lysosomal storage diseases

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

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).

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Enzyme-replacement therapy in a 5-month-old boy with attenuated presymptomatic MPS I: 5-year follow-up

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Enzyme replacement in murine mucopolysaccharidosis type VII: neuronal and glial response to beta-glucuronidase requires early initiation of enzyme replacement therapy.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Placenta analysis of prenatally diagnosed patients reveals early GAG storage in mucopolysaccharidoses II and VI.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Evidence of a progressive motor dysfunction in Mucopolysaccharidosis type I mice

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Recombinant encapsulated cells overexpressing alpha-L-iduronidase correct enzyme deficiency in human mucopolysaccharidosis type I cells

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Pathogenesis of aortic dilatation in mucopolysaccharidosis VII mice may involve complement activation

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Bone marrow derived cells decrease inflammation but not oxidative stress in an experimental model of acute myocardial infarction.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Serial noninvasive assessment of progressive pulmonary hypertension in a rat model

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Progression of multiple behavioral deficits with various ages of onset in a murine model of Hurler syndrome.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Intraperitoneal implant of recombinant encapsulated cells overexpressing alpha-L-iduronidase partially corrects visceral pathology in mucopolysaccharidosis type I mice.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Enzyme-replacement therapy in mucopolysaccharidosis I.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase).

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Lysosomal dysfunction results in altered energy balance

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Cardiac findings after enzyme replacement therapy for mucopolysaccharidosis type I

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Aortic stenosis in a patient with Hurler's syndrome after bone marrow transplantation

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Upregulation of elastase proteins results in aortic dilatation in mucopolysaccharidosis I mice

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Enzyme replacement therapy in feline mucopolysaccharidosis I.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Treatment reduces or stabilizes brain imaging abnormalities in patients with MPS I and II.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Efficacy of recombinant human alpha-L-iduronidase (laronidase) on restricted range of motion of upper extremities in mucopolysaccharidosis type I patients

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

Immune tolerance after long-term enzyme-replacement therapy among patients who have mucopolysaccharidosis I

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.03.005

10.1016/J.YMGME.2013.03.005

7 January 2021

Identifiers

DOI

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3 December 2017

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