Diagnosis and treatment trends in mucopolysaccharidosis I: findings from the MPS I Registry (Q34246168)

31 July 2017

Diagnosis and treatment trends in mucopolysaccharidosis I: findings from the MPS I Registry (English)

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31 July 2017

mucopolysaccharidosis I

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6

Roberto Giugliani

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31 July 2017

Kristin D'Aco

1

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31 July 2017

Lisa Underhill

2

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31 July 2017

Lakshmi Rangachari

3

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31 July 2017

Pamela Arn

4

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31 July 2017

Gerald F Cox

5

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31 July 2017

Torayuki Okuyama

7

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31 July 2017

Frits Wijburg

8

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31 July 2017

Paige Kaplan

9

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31 July 2017

language of work or name

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11 January 2012

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European Journal of Pediatrics

31 July 2017

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31 July 2017

171

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31 July 2017

911-919

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31 July 2017

6

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https://scigraph.springernature.com/pub.10.1007/s00431-011-1644-x

31 July 2017

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cites work

Clinical manifestations and treatment of mucopolysaccharidosis type I patients in Latin America as compared with the rest of the world

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357468

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

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357468

Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I.

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357468

Childhood onset of Scheie syndrome, the attenuated form of mucopolysaccharidosis I.

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357468

Transplant outcomes in mucopolysaccharidoses

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357468

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

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357468

Mucopolysaccharidosis I: management and treatment guidelines

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357468

The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UK

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357468

The clinical outcome of Hurler syndrome after stem cell transplantation.

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357468

Clinical presentation and follow-up of patients with the attenuated phenotype of mucopolysaccharidosis type I.

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357468

Can mucopolysaccharidosis type I disease severity be predicted based on a patient's genotype? A comprehensive review of the literature

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357468

The frequency of lysosomal storage diseases in The Netherlands.

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357468

Prevalence of lysosomal storage disorders

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357468

Hurler syndrome: II. Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. The Storage Disease Collaborative Study Group

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357468

Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357468

Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with hurler syndrome

6 July 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00431-011-1644-X

Ethical issues related to the access to orphan drugs in Brazil: the case of mucopolysaccharidosis type I.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00431-011-1644-X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00431-011-1644-X

Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00431-011-1644-X

Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00431-011-1644-X

Pre-transplant risk factors affecting outcome in Hurler syndrome.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00431-011-1644-X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00431-011-1644-X

High rate of postoperative mortality in patients with mucopolysaccharidosis I: findings from the MPS I Registry

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357468

Early presentation in the mucopolysaccharide disorders.

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357468

Attenuated type I mucopolysaccharidosis in the differential diagnosis of juvenile idiopathic arthritis: a series of 13 patients with Scheie syndrome

28 October 2018

1 reference

12 December 2020

The MPS I registry: design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis Type I

1 reference

12 December 2020

Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure

1 reference

12 December 2020

Successful treatment of severe heart failure in an infant with Hurler syndrome

1 reference

12 December 2020

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

1 reference

12 December 2020

Characterization of surgical procedures in patients with mucopolysaccharidosis type I: findings from the MPS I Registry

1 reference

12 December 2020

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

1 reference

12 December 2020

10.1007/S00431-011-1644-X

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DOI

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31 July 2017

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31 July 2017

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