12 year follow up of enzyme-replacement therapy in two siblings with attenuated mucopolysaccharidosis I: the important role of early treatment (Q35953427)

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English	12 year follow up of enzyme-replacement therapy in two siblings with attenuated mucopolysaccharidosis I: the important role of early treatment	scientific article

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scholarly article

1 reference

Europe PubMed Central

11 August 2017

12 year follow up of enzyme-replacement therapy in two siblings with attenuated mucopolysaccharidosis I: the important role of early treatment (English)

1 reference

Europe PubMed Central

11 August 2017

enzyme replacement therapy

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1 reference

based on heuristic

inferred from title

mucopolysaccharidosis I

1 reference

based on heuristic

inferred from title

object named as

Lorne A Clarke

2

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author name string

Orazio Gabrielli

1

1 reference

Europe PubMed Central

11 August 2017

Anna Ficcadenti

3

1 reference

Europe PubMed Central

11 August 2017

Lucia Santoro

4

1 reference

Europe PubMed Central

11 August 2017

Lucia Zampini

5

1 reference

Europe PubMed Central

11 August 2017

Nicola Volpi

6

1 reference

Europe PubMed Central

11 August 2017

Giovanni V Coppa

7

1 reference

Europe PubMed Central

11 August 2017

publication date

10 March 2016

1 reference

Europe PubMed Central

11 August 2017

BMC Medical Genetics

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Europe PubMed Central

11 August 2017

17

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Europe PubMed Central

11 August 2017

19

1 reference

Europe PubMed Central

11 August 2017

https://scigraph.springernature.com/pub.10.1186/s12881-016-0284-4

0 references

Early treatment with laronidase improves clinical outcomes in patients with attenuated MPS I: a retrospective case series analysis of nine sibships

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4785727

2 October 2017

Early initiation of enzyme replacement therapy for the mucopolysaccharidoses

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4785727

2 October 2017

Therapy for the mucopolysaccharidoses

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4785727

2 October 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4785727

2 October 2017

Development of the EQ-5D-Y: a child-friendly version of the EQ-5D

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4785727

2 October 2017

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=4785727

2 October 2017

Extracellular matrix disruption is an early event in the pathogenesis of skeletal disease in mucopolysaccharidosis I.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4785727

24 September 2018

Does early use of enzyme replacement therapy alter the natural history of mucopolysaccharidosis I? Experience in three siblings

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4785727

24 September 2018

Plasma and urinary levels of dermatan sulfate and heparan sulfate derived disaccharides after long-term enzyme replacement therapy (ERT) in MPS I: correlation with the timing of ERT and with total urinary excretion of glycosaminoglycans.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4785727

24 September 2018

Effect of 6 years of enzyme replacement therapy on plasma and urine glycosaminoglycans in attenuated MPS I patients

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4785727

24 September 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=4785727

24 September 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=4785727

24 September 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=4785727

24 September 2018

Biomarker responses correlate with antibody status in mucopolysaccharidosis type I patients on long-term enzyme replacement therapy

1 reference

https://pubmed.ncbi.nlm.nih.gov/26965916

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/26965916

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Enzyme replacement therapy in an attenuated case of mucopolysaccharidosis type I (Scheie syndrome): a 6.5-year detailed follow-up

1 reference

https://pubmed.ncbi.nlm.nih.gov/26965916

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1186/S12881-016-0284-4

1 reference

Europe PubMed Central

11 August 2017

1 reference

Europe PubMed Central

11 August 2017

1 reference

Europe PubMed Central

11 August 2017

ResearchGate publication ID

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