(Q39663434)

13 September 2017

title

The role of immune tolerance induction in restoration of the efficacy of ERT in Pompe disease (English)

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13 September 2017

Emanuela Lacaná

1

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13 September 2017

Lynne P Yao

series ordinal

2

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13 September 2017

Anne R Pariser

series ordinal

3

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13 September 2017

Amy S Rosenberg

series ordinal

4

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13 September 2017

publication date

17 January 2012

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American Journal of Medical Genetics Part C: Seminars in Medical Genetics

13 September 2017

published in

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13 September 2017

volume

160C

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13 September 2017

issue

1

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13 September 2017

page(s)

30-39

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Glucose tetrasaccharide as a biomarker for monitoring the therapeutic response to enzyme replacement therapy for Pompe disease

13 September 2017

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1 reference

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Induction of immune tolerance using rituximab in a child with severe haemophilia B with inhibitors and anaphylaxis to factor IX.

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Modifying exogenous glucocerebrosidase for effective replacement therapy in Gaucher disease.

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Brain development in infantile-onset Pompe disease treated by enzyme replacement therapy.

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Muscle-specific promoters may be necessary for adeno-associated virus-mediated gene transfer in the treatment of muscular dystrophies.

21 January 2018

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Immunomics: discovering new targets for vaccines and therapeutics

21 January 2018

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Neural deficits contribute to respiratory insufficiency in Pompe disease

21 January 2018

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Mannose 6-phosphate receptors: new twists in the tale

21 January 2018

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Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy

21 January 2018

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Clinical manifestation and natural course of late-onset Pompe's disease in 54 Dutch patients

21 January 2018

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Antibody-mediated regulation of the immune response

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Enhanced endothelial delivery and biochemical effects of α-galactosidase by ICAM-1-targeted nanocarriers for Fabry disease

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A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease

21 January 2018

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

21 January 2018

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Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants

21 January 2018

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Immunomodulatory gene therapy in lysosomal storage disorders

21 January 2018

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Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle

21 January 2018

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Structure and function of the mannose 6-phosphate/insulinlike growth factor II receptors

21 January 2018

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Dilative arteriopathy and basilar artery dolichoectasia complicating late-onset Pompe disease.

21 January 2018

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Glycosylation-independent targeting enhances enzyme delivery to lysosomes and decreases storage in mucopolysaccharidosis type VII mice

21 January 2018

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Late-onset acid maltase deficiency associated with intracranial aneurysm

21 January 2018

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Epoetin-associated pure red cell aplasia: past, present, and future considerations

21 January 2018

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Elimination of antibodies to recombinant enzyme in Pompe's disease.

21 January 2018

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Removal of B cell epitopes as a practical approach for reducing the immunogenicity of foreign protein-based therapeutics

21 January 2018

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Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease.

21 January 2018

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Fc-receptors as regulators of immunity.

21 January 2018

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Deconstructing Pompe disease by analyzing single muscle fibers: to see a world in a grain of sand...

21 January 2018

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CRIM-negative infantile Pompe disease: 42-month treatment outcome.

21 January 2018

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Abnormalities of cerebral arteries are frequent in patients with late-onset Pompe disease.

21 January 2018

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Identification of two subtypes of infantile acid maltase deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FAJMG.C.31316

Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FAJMG.C.31316

Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease

21 January 2018

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A randomized study of alglucosidase alfa in late-onset Pompe's disease

21 January 2018

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https://api.crossref.org/works/10.1002%2FAJMG.C.31316

The natural course of non-classic Pompe's disease; a review of 225 published cases

21 January 2018

1 reference

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Delivery of beta-galactosidase to mouse brain via the blood-brain barrier transferrin receptor

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FAJMG.C.31316

Conjugation of mannose 6-phosphate-containing oligosaccharides to acid alpha-glucosidase improves the clearance of glycogen in pompe mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FAJMG.C.31316

Carbohydrate-remodelled acid alpha-glucosidase with higher affinity for the cation-independent mannose 6-phosphate receptor demonstrates improved delivery to muscles of Pompe mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FAJMG.C.31316

Pompe disease: Design, methodology, and early findings from the Pompe Registry

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FAJMG.C.31316

21 January 2018

Identifiers

DOI

10.1002/AJMG.C.31316

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13 September 2017

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