Enzyme replacement in neuronal storage disorders in the pediatric population.

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Ex vivo gene therapy using patient iPSC-derived NSCs reverses pathology in the brain of a homologous mouse model A Neuron-Specific Gene Therapy Relieves Motor Deficits in Pompe Disease Mice.

P2860

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P2860

Enzyme replacement in neuronal storage disorders in the pediatric population.

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http://www.wikidata.org/entity/Q43948150

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2013 nî lūn-bûn

@nan

2013年の論文

@ja

2013年学术文章

@wuu

2013年学术文章

@zh-cn

2013年学术文章

@zh-hans

2013年学术文章

@zh-my

2013年学术文章

@zh-sg

2013年學術文章

@yue

2013年學術文章

@zh

2013年學術文章

@zh-hant

name

Enzyme replacement in neuronal storage disorders in the pediatric population.

@en

Enzyme replacement in neuronal storage disorders in the pediatric population.

@nl

type

Item

label

Enzyme replacement in neuronal storage disorders in the pediatric population.

@en

Enzyme replacement in neuronal storage disorders in the pediatric population.

@nl

prefLabel

Enzyme replacement in neuronal storage disorders in the pediatric population.

@en

Enzyme replacement in neuronal storage disorders in the pediatric population.

@nl

P1476

Enzyme replacement in neuronal storage disorders in the pediatric population.

@en

P2093

Erika F Augustine

http://www.w3.org/2001/XMLSchema#string

Jonathan W Mink

http://www.w3.org/2001/XMLSchema#string

P2860

Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg

Characterization of gene-activated human acid- -glucosidase: Crystal structure, glycan composition, and internalization into macrophages

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

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

Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease

Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease.

Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry.

Revised recommendations for the management of Gaucher disease in children.

Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double-blind, multinational, Phase 3 study.

Velaglucerase alfa enzyme replacement therapy compared with imiglucerase in patients with Gaucher disease.

P2888

s11940-013-0256-3

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634-651

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scholarly article

P356

10.1007/S11940-013-0256-3

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2013-10-01T00:00:00Z

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1037767434

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Enzyme replacement in neuronal storage disorders in the pediatric population.

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P2860

P2860

Enzyme replacement in neuronal storage disorders in the pediatric population.

description

name

type

label

prefLabel

P1433

P1476

P2093

P2860

P2888

P304

P31

P356

P433

P478

P577

P6179

P698

P356

P1433

P1476

P2093

P2860

P2888

P304

P31

P356

P433

P478

P577

P6179

P698