First human treatment with investigational rhGUS enzyme replacement therapy in an advanced stage MPS VII patient (Q42090139)

16 October 2017

First human treatment with investigational rhGUS enzyme replacement therapy in an advanced stage MPS VII patient (English)

1 reference

enzyme replacement therapy

16 October 2017

0 references

1 reference

Joyce E Fox

1 reference

16 October 2017

Linda Volpe

1 reference

16 October 2017

Josephine Bullaro

1 reference

16 October 2017

Emil D Kakkis

1 reference

16 October 2017

William S Sly

1 reference

16 October 2017

7 November 2014

1 reference

Molecular Genetics and Metabolism

16 October 2017

1 reference

16 October 2017

114

1 reference

16 October 2017

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16 October 2017

203-208

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Chondroitin 6-Sulfate as a Novel Biomarker for Mucopolysaccharidosis IVA and VII.

16 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4360956

Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study

29 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4360956

Disease-specific non-reducing end carbohydrate biomarkers for mucopolysaccharidoses

29 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4360956

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

29 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4360956

Murine mucopolysaccharidosis type VII: the impact of therapies on the clinical course and pathology in a murine model of lysosomal storage disease

29 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4360956

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

29 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4360956

Beta glucuronidase deficiency: report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis

29 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4360956

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

29 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4360956

Enzyme-replacement therapy in mucopolysaccharidosis I.

17 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4360956

Mucopolysaccharidosis VII: postmortem biochemical and pathological findings in a young adult with beta-glucuronidase deficiency.

17 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4360956

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

17 August 2018

1 reference

Crossref

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.10.017

Enzyme replacement with recombinant beta-glucuronidase in murine mucopolysaccharidosis type VII: impact of therapy during the first six weeks of life on subsequent lysosomal storage, growth, and survival

7 January 2021

1 reference

Crossref

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.10.017

10.1016/J.YMGME.2014.10.017

7 January 2021

Identifiers

DOI

1 reference

release_4q7k3emowfgtpexb2os75jg4lm

16 October 2017

1 reference

Fatcat

https://api.fatcat.wiki/v0/release/4q7k3emowfgtpexb2os75jg4lm

mapped directly with Wikidata item

24 November 2022

based on heuristic

1 reference

16 October 2017

PubMed publication ID

25468648

1 reference