Enzyme replacement in neuronal storage disorders in the pediatric population (Q43948150)

28 November 2017

Enzyme replacement in neuronal storage disorders in the pediatric population (English)

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28 November 2017

Erika F Augustine

1

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PubMed ID

23955157

28 November 2017

Jonathan W Mink

series ordinal

2

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28 November 2017

1 October 2013

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Current treatment options in neurology

28 November 2017

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28 November 2017

15

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28 November 2017

5

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28 November 2017

634-651

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https://scigraph.springernature.com/pub.10.1007/s11940-013-0256-3

28 November 2017

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Observational clinical study in juvenile-adult glycogenosis type 2 patients undergoing enzyme replacement therapy for up to 4 years.

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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Enzyme replacement is associated with better cognitive outcomes after transplant in Hurler syndrome

21 January 2018

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21 January 2018

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

21 January 2018

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Idursulfase treatment of Hunter syndrome in children younger than 6 years: results from the Hunter Outcome Survey

21 January 2018

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The long-term international safety experience of imiglucerase therapy for Gaucher disease

21 January 2018

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Management of neutralizing antibody to Ceredase in a patient with type 3 Gaucher disease.

21 January 2018

1 reference

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Lysosomal enzyme can bypass the blood-brain barrier and reach the CNS following intranasal administration

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS11940-013-0256-3

Engineering a lysosomal enzyme with a derivative of receptor-binding domain of apoE enables delivery across the blood-brain barrier

21 January 2018

1 reference

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Rapid deterioration of a patient with mucopolysaccharidosis type I during interruption of enzyme replacement therapy

21 January 2018

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12 December 2020

Long-term observational, non-randomized study of enzyme replacement therapy in late-onset glycogenosis type II

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12 December 2020

Enzyme replacement therapy with agalsidase alfa in children with Fabry disease

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12 December 2020

Enzyme replacement therapy in type III Gaucher disease

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12 December 2020

Enzyme replacement therapy for Fabry disease: some answers but more questions

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12 December 2020

Management of neuronopathic Gaucher disease: revised recommendations

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12 December 2020

Nephrotic syndrome complicating alpha-glucosidase replacement therapy for Pompe disease

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12 December 2020

Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial

1 reference

12 December 2020

A phase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome)

1 reference

12 December 2020

Human acid alpha-glucosidase from rabbit milk has therapeutic effect in mice with glycogen storage disease type II

1 reference

12 December 2020

Booster-effect with velaglucerase alfa in patients with Gaucher disease switched from long-term imiglucerase therapy: early Access Program results from Jerusalem

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12 December 2020

Fabry disease in children and response to enzyme replacement therapy: results from the Fabry Outcome Survey

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12 December 2020

Agalsidase-beta therapy for advanced Fabry disease: a randomized trial

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12 December 2020

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

1 reference

12 December 2020

Neurological outcome of a patient with Gaucher disease type III treated by enzymatic replacement therapy

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12 December 2020

Gaucher disease: enzyme therapy in the acute neuronopathic variant

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12 December 2020

Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome

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12 December 2020

10.1007/S11940-013-0256-3

Identifiers

DOI

1 reference

PubMed ID

23955157

Dimensions Publication ID

28 November 2017

0 references

1 reference

PubMed ID

23955157