Current and potential therapeutic strategies for mucopolysaccharidoses (Q38194635)

30 August 2017

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title

Current and potential therapeutic strategies for mucopolysaccharidoses (English)

1 reference

30 August 2017

H Noh

1

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PubMed ID

24612142

30 August 2017

J I Lee

series ordinal

2

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30 August 2017

25 February 2014

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Journal of Clinical Pharmacy and Therapeutics

30 August 2017

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30 August 2017

39

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30 August 2017

215-224

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30 August 2017

3

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The mucopolysaccharidoses: a clinical review and guide to management

30 August 2017

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Overview of the mucopolysaccharidoses

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Diagnosis and treatment trends in mucopolysaccharidosis I: findings from the MPS I Registry

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Mucopolysaccharidosis type II in females and response to enzyme replacement therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Initial report from the Hunter Outcome Survey

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome).

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Sanfilippo syndrome: a mini-review

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Cell- and gene-based therapeutic approaches for neurological deficits in mucopolysaccharidoses

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Genetic heterogeneity and clinical variability in the Sanfilippo syndrome (types A, B, and C)

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Mucopolysaccharidosis type IVA (Morquio A disease): clinical review and current treatment

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

International Morquio A Registry: clinical manifestation and natural course of Morquio A disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Management guidelines for mucopolysaccharidosis VI.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Mucopolysaccharidosis VI.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Mutations and polymorphisms in GUSB gene in mucopolysaccharidosis VII (Sly Syndrome).

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Long circulating enzyme replacement therapy rescues bone pathology in mucopolysaccharidosis VII murine model

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Clinical and biochemical manifestations of hyaluronidase deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

The structure of glycosaminoglycans and their interactions with proteins.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

The mucopolysaccharidoses: a success of molecular medicine.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Pathogenic cascades in lysosomal disease-Why so complex?

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Perineuronal nets potentially protect against oxidative stress

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Pathogenic mechanisms in lysosomal disease: a reappraisal of the role of the lysosome

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Common and uncommon pathogenic cascades in lysosomal storage diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Differential subcellular localization of cholesterol, gangliosides, and glycosaminoglycans in murine models of mucopolysaccharide storage disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Secondary accumulation of gangliosides in lysosomal storage disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Gangliosides as modulators of dendritogenesis in normal and storage disease-affected pyramidal neurons.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Gangliosides as apoptotic signals in ER stress response

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Enzyme replacement therapy for the mucopolysaccharide storage disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Treatment of lysosomal storage disorders : progress with enzyme replacement therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

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

https://api.crossref.org/works/10.1111%2FJCPT.12136

Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human alpha-L-iduronidase (laronidase).

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Mucopolysaccharidosis I: management and treatment guidelines

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

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/10.1111%2FJCPT.12136

Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

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/10.1111%2FJCPT.12136

Transplant outcomes in mucopolysaccharidoses

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Long-term outcome of Hurler syndrome following bone marrow transplantation.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Bone marrow transplantation in children with Hunter syndrome: outcome after 7 to 17 years.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Bone marrow transplantation in Hunter syndrome.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Therapy for the mucopolysaccharidoses

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Orthopaedic aspects of mucopolysaccharidoses

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Correction of CNS defects in the MPSII mouse model via systemic enzyme replacement therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Impact of high-dose, chemically modified sulfamidase on pathology in a murine model of MPS IIIA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Intracerebral injection of sulfamidase delays neuropathology in murine MPS-IIIA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Intrathecal recombinant human 4-sulfatase reduces accumulation of glycosaminoglycans in dura of mucopolysaccharidosis VI cats.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

CNS penetration of intrathecal-lumbar idursulfase in the monkey, dog and mouse: implications for neurological outcomes of lysosomal storage disorder

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Reversal of lysosomal storage in brain of adult MPS-I mice with intravenous Trojan horse-iduronidase fusion protein

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Enhancement of drug delivery: enzyme-replacement therapy for murine Morquio A syndrome.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Inhibition of glycosaminoglycan synthesis using rhodamine B in a mouse model of mucopolysaccharidosis type IIIA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Improvement in behaviour after substrate deprivation therapy with rhodamine B in a mouse model of MPS IIIA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Substrate reduction therapies for mucopolysaccharidoses

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Genistein-mediated inhibition of glycosaminoglycan synthesis, which corrects storage in cells of patients suffering from mucopolysaccharidoses, acts by influencing an epidermal growth factor-dependent pathway.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Genistein reduces glycosaminoglycan levels in a mouse model of mucopolysaccharidosis type II

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Genistein reduces lysosomal storage in peripheral tissues of mucopolysaccharide IIIB mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Genistein improves neuropathology and corrects behaviour in a mouse model of neurodegenerative metabolic disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Genistin-rich soy isoflavone extract in substrate reduction therapy for Sanfilippo syndrome: An open-label, pilot study in 10 pediatric patients

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

The role of the iminosugar N-butyldeoxynojirimycin (miglustat) in the management of type I (non-neuronopathic) Gaucher disease: a position statement

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Sustained production of beta-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Widespread biochemical correction of murine mucopolysaccharidosis type VII pathology by liver hydrodynamic plasmid delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Correction of pathological accumulation of glycosaminoglycans in central nervous system and peripheral tissues of MPSIIIA mice through systemic AAV9 gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Gene therapy for mucopolysaccharidosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Incidence of the mucopolysaccharidoses in Northern Ireland

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UK

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

Incidence of inborn errors of metabolism in British Columbia, 1969-1996.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCPT.12136

21 January 2018

Identifiers

DOI

10.1111/JCPT.12136

1 reference

30 August 2017

1 reference

PubMed ID

24612142