(Q36302871)

13 August 2017

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title

Current and emerging therapies for the lysosomal storage disorders (English)

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13 August 2017

Gregory M Pastores

1

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13 August 2017

Natalie L Barnett

series ordinal

2

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13 August 2017

language of work or name

English

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publication date

1 November 2005

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Expert Opinion on Emerging Drugs

13 August 2017

published in

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13 August 2017

volume

10

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13 August 2017

issue

4

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13 August 2017

page(s)

891-902

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Lysosomal storage disorders

13 August 2017

cites work

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

Glycosphingolipidoses: beyond the enzymatic defect

21 January 2018

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Specific induction of macrophage inflammatory protein 1-alpha in glial cells of Sandhoff disease model mice associated with accumulation of N-acetylhexosaminyl glycoconjugates

21 January 2018

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Divergent phenotypes in Gaucher disease implicate the role of modifiers

21 January 2018

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https://api.crossref.org/works/10.1517%2F14728214.10.4.891

Direct multiplex assay of lysosomal enzymes in dried blood spots for newborn screening

21 January 2018

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A genetic profile of contemporary Jewish populations

21 January 2018

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Quality of life of patients with Fabry disease.

21 January 2018

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https://api.crossref.org/works/10.1517%2F14728214.10.4.891

Assessment of health-related quality-of-life in males with Anderson Fabry Disease before therapeutic intervention

21 January 2018

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Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 98 hemizygous males

21 January 2018

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Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 60 obligate carrier females

21 January 2018

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Musculoskeletal complications associated with lysosomal storage disorders: Gaucher disease and Hurler-Scheie syndrome (mucopolysaccharidosis type I).

21 January 2018

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Pharmacological treatment of nephropathic cystinosis with cysteamine

21 January 2018

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https://api.crossref.org/works/10.1517%2F14728214.10.4.891

Therapeutic apheresis in renal and metabolic diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

Allogeneic stem cell transplantation for the treatment of lysosomal and peroxisomal metabolic diseases

21 January 2018

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

21 January 2018

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Microglia: The Effector Cell for Reconstitution of the Central Nervous System following Bone Marrow Transplantation for Lysosomal and Peroxisomal Storage Diseases

21 January 2018

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Long-term follow-up following bone marrow transplantation for Hunter disease.

21 January 2018

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https://api.crossref.org/works/10.1517%2F14728214.10.4.891

The current status of hematopoietic cell transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

Recombinant protein expression for therapeutic applications

21 January 2018

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https://api.crossref.org/works/10.1517%2F14728214.10.4.891

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

21 January 2018

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The mannose receptor family

21 January 2018

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Deterioration of the auditory brainstem response in children with type 3 Gaucher disease.

21 January 2018

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Enzyme replacement therapy in Fabry disease: a randomized controlled trial

21 January 2018

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Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry's disease

21 January 2018

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Advances in the management of Anderson-Fabry disease: enzyme replacement therapy

21 January 2018

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Enzyme replacement reverses abnormal cerebrovascular responses in Fabry disease

21 January 2018

1 reference

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Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase).

21 January 2018

1 reference

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Direct comparison of measures of endurance, mobility, and joint function during enzyme-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): results after 48 weeks in a phase 2 open-label clinical study of recombinant human N-ac

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

Safety and efficacy of recombinant acid alpha-glucosidase (rhGAA) in patients with classical infantile Pompe disease: results of a phase II clinical trial

21 January 2018

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21 January 2018

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Sustained therapeutic effects of oral miglustat (Zavesca, N-butyldeoxynojirimycin, OGT 918) in type I Gaucher disease.

21 January 2018

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Substrate reduction therapy: miglustat as a remedy for symptomatic patients with Gaucher disease type 1.

21 January 2018

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Blood to brain to the rescue.

21 January 2018

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https://api.crossref.org/works/10.1517%2F14728214.10.4.891

Agents for the treatment of glycosphingolipid storage disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

Enzyme replacement and enhancement therapies for lysosomal diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

Modulation of neurodegeneration by molecular chaperones

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

Improvement in cardiac function in the cardiac variant of Fabry's disease with galactose-infusion therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

Chemical chaperones increase the cellular activity of N370S beta -glucosidase: a therapeutic strategy for Gaucher disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

Chemical chaperone therapy for brain pathology in G(M1)-gangliosidosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

Pharmacological enhancement of beta-hexosaminidase activity in fibroblasts from adult Tay-Sachs and Sandhoff Patients.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

A synthetic chaperone corrects the trafficking defect and disease phenotype in a protein misfolding disorder

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

Effective gene therapy for an inherited CNS disease in a large animal model

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

Bone marrow stem cell gene therapy of arylsulfatase A-deficient mice, using an arylsulfatase A mutant that is hypersecreted from retrovirally transduced donor-type cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

Muscle as a putative producer of acid alpha-glucosidase for glycogenosis type II gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

Feasibility of gene therapy for late neuronal ceroid lipofuscinosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

Sphingolipids involved in the induction of multinuclear cell formation.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

Delivery of therapeutic agents to the central nervous system: the problems and the possibilities.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

The mousetrap: what we can learn when the mouse model does not mimic the human disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

NSAIDs increase survival in the Sandhoff disease mouse: synergy with N-butyldeoxynojirimycin

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728214.10.4.891

10.1517/14728214.10.4.891

21 January 2018

Identifiers

DOI

1 reference

13 August 2017

1 reference