Early initiation of enzyme replacement therapy for the mucopolysaccharidoses (Q38175637)

30 August 2017

Early initiation of enzyme replacement therapy for the mucopolysaccharidoses (English)

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enzyme replacement therapy

30 August 2017

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author name string

Joseph Muenzer

series ordinal

1

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PubMed ID

24388732

30 August 2017

language of work or name

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11 December 2013

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Molecular Genetics and Metabolism

30 August 2017

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30 August 2017

111

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30 August 2017

63-72

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30 August 2017

2

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Mucopolysaccharidosis type II in females: case report and review of literature.

30 August 2017

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Expression of the disease on female carriers of X-linked lysosomal disorders: a brief review

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

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.2013.11.015

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.2013.11.015

Enzyme replacement therapy for mucopolysaccharidosis VI: A phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, o

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Idursulfase treatment of Hunter syndrome in children younger than 6 years: results from the Hunter Outcome Survey

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

The role of enzyme replacement therapy in severe Hunter syndrome-an expert panel consensus

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Laronidase (Aldurazyme): enzyme replacement therapy for mucopolysaccharidosis type I

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Mucopolysaccharidosis I: management and treatment guidelines

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Enzyme replacement is associated with better cognitive outcomes after transplant in Hurler syndrome

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Mucopolysaccharidosis type III (Sanfilippo Syndrome): emerging treatment strategies

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Cardiac findings after enzyme replacement therapy for mucopolysaccharidosis type I

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

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.2013.11.015

The natural course and the impact of therapies of cardiac involvement in the mucopolysaccharidoses

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Japan Elaprase Treatment (JET) study: idursulfase enzyme replacement therapy in adult patients with attenuated Hunter syndrome (Mucopolysaccharidosis II, MPS II).

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Mucopolysaccharidosis: cardiologic features and effects of enzyme-replacement therapy in 24 children with MPS I, II and VI.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme®) therapy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Brain and spine MRI features of Hunter disease: frequency, natural evolution and response to therapy.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

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.2013.11.015

Lung function testing: selection of reference values and interpretative strategies. American Thoracic Society

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Reference ranges for spirometry across all ages: a new approach

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

A retrospective audit of anesthetic techniques and complications in children with mucopolysaccharidoses

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Inborn errors of mucopolysaccharide metabolism

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Pathogenic cascades in lysosomal disease-Why so complex?

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Pathogenesis of skeletal and connective tissue involvement in the mucopolysaccharidoses: glycosaminoglycan storage is merely the instigator

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Mechanism of glycosaminoglycan-mediated bone and joint disease: implications for the mucopolysaccharidoses and other connective tissue diseases

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

The molecular diversity of glycosaminoglycans shapes animal development.

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Glycosaminoglycans and their proteoglycans: host-associated molecular patterns for initiation and modulation of inflammation

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

The mucopolysaccharidoses: a success of molecular medicine.

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Heparan sulphate proteoglycans fine-tune mammalian physiology

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Endogenous heparan sulfate and heparin modulate bone morphogenetic protein-4 signaling and activity

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

DNA damage in leukocytes from pretreatment mucopolysaccharidosis type II patients; protective effect of enzyme replacement therapy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Mutational and oxidative stress analysis in patients with mucopolysaccharidosis type I undergoing enzyme replacement therapy.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Glycosaminoglycan Chains Affect Exocytic and Endocytic Protein Traffic

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Self-eating in skeletal development: implications for lysosomal storage disorders

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Secondary storage of dermatan sulfate in Sanfilippo disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Secondary accumulation of gangliosides in lysosomal storage disorders

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Secondary lipid accumulation in lysosomal disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Storage problems in lysosomal diseases

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

The heart in the Hurler syndrome: gross, histologic and ultrastructural observations in five necropsy cases

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Sudden death in Hunter syndrome caused by complete atrioventricular block

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Clear cells in the atrioventricular valves of infants with severe human mucopolysaccharidosis (Hurler syndrome) are activated valvular interstitial cells

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

The emerging role of valve interstitial cell phenotypes in regulating heart valve pathobiology

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Extracellular matrix remodelling in human aortic valve disease: the role of matrix metalloproteinases and their tissue inhibitors

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Mucopolysaccharidosis I under enzyme replacement therapy with laronidase--a mortality case with autopsy report

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Histomorphometric analysis of the tibial growth plate in a feline model of mucopolysaccharidosis type VI

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Cartilage and chondrocyte pathology in the mucopolysaccharidoses: The role of glycosaminoglycan-mediated inflammation

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Articular chondrocytes from animals with a dermatan sulfate storage disease undergo a high rate of apoptosis and release nitric oxide and inflammatory cytokines: a possible mechanism underlying degenerative joint disease in the mucopolysaccharidoses

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Involvement of the Toll-like receptor 4 pathway and use of TNF-alpha antagonists for treatment of the mucopolysaccharidoses

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Anti-TNF-alpha therapy enhances the effects of enzyme replacement therapy in rats with mucopolysaccharidosis type VI

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Skeletal dysplasias and their effect on the respiratory system

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Respiratory manifestations in mucopolysaccharidoses

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Upper airway obstructive disease in mucopolysaccharidoses: polysomnography, computed tomography and nasal endoscopy findings.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Airway changes in children with mucopolysaccharidoses.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Narrow trachea in mucopolysaccharidoses

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Skeletal dysplasias: evaluation with impulse oscillometry and thoracoabdominal motion analysis

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Prenatal mucopolysaccharidosis II (Hunter): a pathogenetic study

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

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.2013.11.015

Enzyme replacement therapy from birth in a feline model of mucopolysaccharidosis type VI.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

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.2013.11.015

Enzyme replacement therapy for mucopolysaccharidosis VI from 8 weeks of age--a sibling control study

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Enzyme replacement therapy for mucopolysaccharidosis II from 3 months of age: a 3-year follow-up

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Effects of idursulfase enzyme replacement therapy for Mucopolysaccharidosis type II when started in early infancy: comparison in two siblings

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

Initial report from the Hunter Outcome Survey

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

First experience of enzyme replacement therapy with idursulfase in Spanish patients with Hunter syndrome under 5 years of age: case observations from the Hunter Outcome Survey (HOS).

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2013.11.015

10.1016/J.YMGME.2013.11.015

7 January 2021

Identifiers

DOI

1 reference

30 August 2017

1 reference

PubMed ID

24388732