(Q34304968)

1 August 2017

The natural history of MPS I: global perspectives from the MPS I Registry (English)

1 reference

1 August 2017

3

Roberto Giugliani

1 reference

1 August 2017

Michael Beck

1

1 reference

1 August 2017

Pamela Arn

2

1 reference

1 August 2017

Joseph Muenzer

4

1 reference

1 August 2017

Torayuki Okuyama

5

1 reference

1 August 2017

John Taylor

6

1 reference

1 August 2017

Shari Fallet

7

1 reference

1 August 2017

language of work or name

English

0 references

publication date

27 March 2014

1 reference

1 August 2017

1 reference

1 August 2017

16

1 reference

1 August 2017

10

1 reference

1 August 2017

759-765

1 reference

https://scigraph.springernature.com/pub.10.1038/gim.2014.25

1 August 2017

0 references

cites work

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4189384

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=4189384

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=4189384

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).

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4189384

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=4189384

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=4189384

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4189384

Problems in the use of large data sets to assess effectiveness

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4189384

Enzyme-replacement therapy in mucopolysaccharidosis I.

6 July 2018

1 reference

https://api.crossref.org/works/10.1038%2FGIM.2014.25

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGIM.2014.25

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGIM.2014.25

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGIM.2014.25

Comparison of outcomes research with clinical trials using preexisting data.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGIM.2014.25

Source document verification in the Mucopolysaccharidosis Type I Registry

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGIM.2014.25

Unique frequency of known mutations in Brazilian MPS I patients

21 January 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/24675674

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

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

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/24675674

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

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

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/24675674

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1038/GIM.2014.25

1 reference

1 August 2017

1 reference

1 August 2017

1 reference