(Q36082084)

12 August 2017

1 reference

title

The first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I. (English)

1 reference

enzyme replacement therapy

12 August 2017

0 references

1 reference

James Edward Wraith

1

1 reference

PubMed ID

15794739

12 August 2017

language of work or name

English

0 references

publication date

1 March 2005

1 reference

Expert Opinion on Pharmacotherapy

12 August 2017

1 reference

12 August 2017

6

1 reference

12 August 2017

3

1 reference

12 August 2017

489-506

1 reference

Advances in the treatment of lysosomal storage disease

12 August 2017

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

Alpha-L-iduronidase and enzyme replacement therapy for mucopolysaccharidosis I.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

Enzyme replacement therapy for the mucopolysaccharide storage disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

Mutational analysis of 85 mucopolysaccharidosis type I families: frequency of known mutations, identification of 17 novel mutations and in vitro expression of missense mutations.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

Brain MRI findings in patients with mucopolysaccharidosis types I and II and mild clinical presentation.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

Surgical treatment of carpal tunnel syndrome and trigger digits in children with mucopolysaccharide storage disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

Airway management in mucopolysaccharide storage disorders.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

The eye as a window to inborn errors of metabolism

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

Otologic and laryngologic manifestations of mucopolysaccharidoses after bone marrow transplantation.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

Selective urinary screening for mucopolysaccharidoses.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

Diagnosis of mucopolysaccharidoses: how to avoid false positives and false negatives.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

46 Enzymic diagnosis of the genetic mucopolysaccharide storage disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

Lysosomal storage disorders: emerging therapeutic options require early diagnosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

Overexpression of the human lysosomal enzyme alpha-L-iduronidase in Chinese hamster ovary cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

Enzyme-replacement therapy in mucopolysaccharidosis I.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

Immune tolerance after long-term enzyme-replacement therapy among patients who have mucopolysaccharidosis I

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

Identification and molecular characterization of alpha-L-iduronidase mutations present in mucopolysaccharidosis type I patients undergoing enzyme replacement therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

The 6-min walk distance: change over time and value as a predictor of survival in severe COPD.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

Cord-blood transplants from unrelated donors in patients with Hurler's syndrome

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

Outcome of 27 patients with Hurler's syndrome transplanted from either related or unrelated haematopoietic stem cell sources

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

Physical performance testing in mucopolysaccharidosis I: a pilot study

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14656566.6.3.489

21 January 2018

Identifiers

DOI

10.1517/14656566.6.3.489

1 reference

12 August 2017

1 reference

PubMed ID

15794739