Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure (Q34207169)

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

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scholarly article

1 reference

Europe PubMed Central

31 July 2017

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

1 reference

Europe PubMed Central

31 July 2017

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enzyme replacement therapy

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mucopolysaccharidosis I

9 references

12 March 2019

14 March 2019

21 March 2019

24 March 2019

22 April 2019

25 April 2019

17 June 2019

25 June 2019

based on heuristic

inferred from title

stem cell transplantation

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based on heuristic

inferred from title

object named as

Nizar Mahlaoui

6

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object named as

Robert F Wynn

15

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Jaap J. Boelens

object named as

Jaap J Boelens

2

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Martin Offringa

object named as

Martin Offringa

8

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Vassili Valayannopoulos

13

object named as

Vassili Valayannopoulos

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Europe PubMed Central

31 July 2017

7

object named as

Eugen Mengel

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Europe PubMed Central

31 July 2017

author name string

Minke H de Ru

1

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Europe PubMed Central

31 July 2017

Anibh M Das

3

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Europe PubMed Central

31 July 2017

Simon A Jones

4

1 reference

Europe PubMed Central

31 July 2017

Johanna H van der Lee

5

1 reference

Europe PubMed Central

31 July 2017

Anne O'Meara

9

1 reference

Europe PubMed Central

31 July 2017

Rossella Parini

10

1 reference

Europe PubMed Central

31 July 2017

Attilio Rovelli

11

1 reference

Europe PubMed Central

31 July 2017

Karl-Walter Sykora

12

1 reference

Europe PubMed Central

31 July 2017

Ashok Vellodi

14

1 reference

Europe PubMed Central

31 July 2017

Frits A Wijburg

16

1 reference

Europe PubMed Central

31 July 2017

publication date

10 August 2011

10 references

Europe PubMed Central

31 July 2017

12 March 2019

14 March 2019

21 March 2019

24 March 2019

22 April 2019

25 April 2019

17 June 2019

25 June 2019

3 July 2021

Orphanet Journal of Rare Diseases

10 references

Europe PubMed Central

31 July 2017

12 March 2019

14 March 2019

21 March 2019

24 March 2019

22 April 2019

25 April 2019

17 June 2019

25 June 2019

3 July 2021

6

1 reference

Europe PubMed Central

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55

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Europe PubMed Central

31 July 2017

copyright license

Creative Commons Attribution 2.0 Generic

9 references

Europe PubMed Central

12 March 2019

Europe PubMed Central

14 March 2019

Europe PubMed Central

21 March 2019

Europe PubMed Central

24 March 2019

Europe PubMed Central

22 April 2019

Europe PubMed Central

25 April 2019

Europe PubMed Central

17 June 2019

Europe PubMed Central

25 June 2019

Europe PubMed Central

3 July 2021

copyright status

0 references

https://scigraph.springernature.com/pub.10.1186/1750-1172-6-55

0 references

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

5 July 2018

Current international perspectives on hematopoietic stem cell transplantation for inherited metabolic disorders.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

5 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

5 July 2018

Weighted kappa: nominal scale agreement with provision for scaled disagreement or partial credit

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

5 July 2018

A Delphi clinical practice protocol for the management of very long chain acyl-CoA dehydrogenase deficiency

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

5 July 2018

Mucopolysaccharidosis I: management and treatment guidelines

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

5 July 2018

Tandem mass spectrometry for the direct assay of lysosomal enzymes in dried blood spots: application to screening newborns for mucopolysaccharidosis I

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

5 July 2018

Incidence and prevalence of mucopolysaccharidosis type 1 in the Irish republic

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

5 July 2018

A Delphi-based consensus clinical practice protocol for the diagnosis and management of 3-methylcrotonyl CoA carboxylase deficiency

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

5 July 2018

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).

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

5 July 2018

Trends in haematopoietic cell transplantation for inborn errors of metabolism

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

5 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

5 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

5 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

5 July 2018

Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

5 July 2018

Multiple polymorphisms within the alpha-L-iduronidase gene (IDUA): implications for a role in modification of MPS-I disease phenotype

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

5 July 2018

Identification of mutations in the alpha-L-iduronidase gene (IDUA) that cause Hurler and Scheie syndromes

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

5 July 2018

IDUA mutational profiling of a cohort of 102 European patients with mucopolysaccharidosis type I: identification and characterization of 35 novel α-L-iduronidase (IDUA) alleles.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

28 October 2018

Laronidase for cardiopulmonary disease in Hurler syndrome 12 years after bone marrow transplantation.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

28 October 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

28 October 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

28 October 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

28 October 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

28 October 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

28 October 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

28 October 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

28 October 2018

Enzyme-replacement therapy in mucopolysaccharidosis I.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

28 October 2018

Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3170181

28 October 2018

Cumulative incidence rates of the mucopolysaccharidoses in Germany

1 reference

https://pubmed.ncbi.nlm.nih.gov/21831279

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

https://pubmed.ncbi.nlm.nih.gov/21831279

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

https://pubmed.ncbi.nlm.nih.gov/21831279

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

https://pubmed.ncbi.nlm.nih.gov/21831279

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

https://pubmed.ncbi.nlm.nih.gov/21831279

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1186/1750-1172-6-55

1 reference

Europe PubMed Central

31 July 2017

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1 reference

Europe PubMed Central

31 July 2017

1 reference

Europe PubMed Central

31 July 2017

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