Therapeutic approaches to bone pathology in Gaucher disease: past, present and future (Q37926030)

25 August 2017

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title

Therapeutic approaches to bone pathology in Gaucher disease: past, present and future (English)

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25 August 2017

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Ozlem Goker-Alpan

1

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25 August 2017

11 August 2011

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Molecular Genetics and Metabolism

25 August 2017

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25 August 2017

104

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25 August 2017

438-447

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25 August 2017

4

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Phenotypic continuum in neuronopathic Gaucher disease: an intermediate phenotype between type 2 and type 3.

25 August 2017

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Twin pairs showing discordance of phenotype in adult Gaucher's disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Gaucher disease: variability in phenotype among siblings

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Divergent phenotypes in Gaucher disease implicate the role of modifiers

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Reactions of vessel walls and brain parenchyma to the accumulation of Gaucher cells in the Norrbottnian type (type III) of Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Skeletal aspects of Gaucher disease: a review

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Imiglucerase (Cerezyme) improves quality of life in patients with skeletal manifestations of Gaucher disease.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Early achievement and maintenance of the therapeutic goals using velaglucerase alfa in type 1 Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

A plant-derived recombinant human glucocerebrosidase enzyme--a preclinical and phase I investigation

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Osseous manifestations of adult Gaucher disease in the era of enzyme replacement therapy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Therapeutic goals in the treatment of Gaucher disease.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Type 1 Gaucher disease: significant disease manifestations in "asymptomatic" homozygotes

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Timing of initiation of enzyme replacement therapy after diagnosis of type 1 Gaucher disease: effect on incidence of avascular necrosis

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Osteopenia in Gaucher disease develops early in life: response to imiglucerase enzyme therapy in children, adolescents and adults

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Osteoclastic bone resorption induced by innate immune responses

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Origin of osteoclasts: mature monocytes and macrophages are capable of differentiating into osteoclasts under a suitable microenvironment prepared by bone marrow-derived stromal cells

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Osteoclasts have multiple roles in bone in addition to bone resorption

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

The Gaucher registry: demographics and disease characteristics of 1698 patients with Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Impact of imiglucerase on the serum glycosylated-ferritin level in Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Correlation of MRI-Based bone marrow burden score with genotype and spleen status in Gaucher's disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

The underrecognized progressive nature of N370S Gaucher disease and assessment of cancer risk in 403 patients

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Bone density in Type 1 Gaucher disease.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Skeletal involvement in Gaucher's disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Bone and joint complications related to Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

A benchmark analysis of the achievement of therapeutic goals for type 1 Gaucher disease patients treated with imiglucerase

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

The female Gaucher patient: the impact of enzyme replacement therapy around key reproductive events (menstruation, pregnancy and menopause).

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

The management of pregnancy in Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Pregnancies in Gaucher disease: a 5-year study

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Orthopedic considerations in Gaucher disease since the advent of enzyme replacement therapy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Glucocerebrosidase gene-deficient mouse recapitulates Gaucher disease displaying cellular and molecular dysregulation beyond the macrophage

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Vertebral fractures in Gaucher disease type I: data from the French "Observatoire" on Gaucher disease (FROG).

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

The clinical and demographic characteristics of nonneuronopathic Gaucher disease in 887 children at diagnosis

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Bone complications in children with Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Differential effects of enzyme supplementation therapy on manifestations of type 1 Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Marked elevation of plasma chitotriosidase activity. A novel hallmark of Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

CCL18: a urinary marker of Gaucher cell burden in Gaucher patients

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Biomarkers for lysosomal storage disorders: identification and application as exemplified by chitotriosidase in Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Increased incidence of cancer in adult Gaucher disease in Western Europe

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Gaucher disease and cancer incidence: a study from the Gaucher Registry.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Pro-inflammatory cytokines and the pathogenesis of Gaucher's disease: increased release of interleukin-6 and interleukin-10.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Cytokine mRNA in Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Tumor necrosis factor-alpha increases circulating osteoclast precursor numbers by promoting their proliferation and differentiation in the bone marrow through up-regulation of c-Fms expression.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Markers of bone turnover in Gaucher disease: modeling the evolution of bone disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Osteoblasts and osteoclasts in bone remodeling and inflammation

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Gaucher disease and bone: laboratory and skeletal mineral density variations during a long period of enzyme replacement therapy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Characterization of Gaucher disease bone marrow mesenchymal stromal cells reveals an altered inflammatory secretome

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Individualization of long-term enzyme replacement therapy for Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Evidence-based recommendations for monitoring bone disease and the response to enzyme replacement therapy in Gaucher patients

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Management of non-neuronopathic Gaucher disease with special reference to pregnancy, splenectomy, bisphosphonate therapy, use of biomarkers and bone disease monitoring

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Quantitative chemical shift imaging of vertebral bone marrow in patients with Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Quantification of skeletal involvement in adults with type I Gaucher's disease: fat fraction measured by Dixon quantitative chemical shift imaging as a valid parameter

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Dixon quantitative chemical shift imaging is a sensitive tool for the evaluation of bone marrow responses to individualized doses of enzyme supplementation therapy in type 1 Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Quantification of bone involvement in Gaucher disease: MR imaging bone marrow burden score as an alternative to Dixon quantitative chemical shift MR imaging--initial experience

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

[MRI bone marrow findings in 63 patients with type I Gaucher disease]

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Use of plain radiography to optimize skeletal outcomes in children with type 1 Gaucher disease in Brazil

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Recommendations for the assessment and monitoring of skeletal manifestations in children with Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Clinical consequences of interrupting enzyme replacement therapy in children with type 1 Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Response of Gaucher bone disease to enzyme replacement therapy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Correlation of bone marrow response with hematological, biochemical, and visceral responses to enzyme replacement therapy of nonneuronopathic (type 1) Gaucher disease in 30 adult patients.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Semiquantitative assessment of skeletal response to enzyme replacement therapy for Gaucher's disease using the bone marrow burden score.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Magnetic resonance imaging of bone marrow changes in Gaucher disease during enzyme replacement therapy: first German long-term results

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Effect of enzyme replacement therapy with imiglucerase on BMD in type 1 Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Improvement of bone disease by imiglucerase (Cerezyme) therapy in patients with skeletal manifestations of type 1 Gaucher disease: results of a 48-month longitudinal cohort study

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Eight-year clinical outcomes of long-term enzyme replacement therapy for 884 children with Gaucher disease type 1.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Decreased bone density in splenectomized Gaucher patients receiving enzyme replacement therapy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Bone density changes with enzyme therapy for Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Low-dose high-frequency enzyme replacement therapy prevents fractures without complete suppression of painful bone crises in patients with severe juvenile onset type I Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Gaucher disease: new molecular approaches to diagnosis and treatment

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

The clinical course of treated and untreated Gaucher disease. A study of 45 patients

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Severe skeletal complications in Japanese patients with type 1 Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Bone marrow response in treated patients with Gaucher disease: evaluation by T1-weighted magnetic resonance images and correlation with reduction in liver and spleen volume

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Effect of low-dose enzyme replacement therapy on bones in Gaucher disease patients with severe skeletal involvement

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

The effect of enzyme replacement therapy on bone crisis and bone pain in patients with type 1 Gaucher disease.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Low frequency maintenance therapy with imiglucerase in adult type I Gaucher disease: a prospective randomized controlled trial.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Enzyme therapy for the treatment of type 1 Gaucher disease: clinical outcomes and dose - response relationships

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

A randomized trial comparing the efficacy and safety of imiglucerase (Cerezyme) infusions every 4 weeks versus every 2 weeks in the maintenance therapy of adult patients with Gaucher disease type 1

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Low-dose high-frequency enzyme replacement therapy for very young children with severe Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

A less costly regimen of alglucerase to treat Gaucher's disease.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

The outcome of clinical parameters in adults with severe Type I Gaucher disease using very low dose enzyme replacement therapy.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Type I Gaucher disease in children with and without enzyme therapy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

The clinical course of untreated Gaucher disease in 22 patients over 10 years: hematological and skeletal manifestations

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Superior effects of high-dose enzyme replacement therapy in type 1 Gaucher disease on bone marrow involvement and chitotriosidase levels: a 2-center retrospective analysis

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Persistent bone disease in adult type 1 Gaucher disease despite increasing doses of enzyme replacement therapy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Pathologic fractures may develop in Gaucher patients receiving enzyme replacement therapy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Production of glucocerebrosidase with terminal mannose glycans for enzyme replacement therapy of Gaucher's disease using a plant cell system

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Effect of mannose chain length on targeting of glucocerebrosidase for enzyme replacement therapy of Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Significant and continuous improvement in bone mineral density among type 1 Gaucher disease patients treated with velaglucerase alfa: 69-month experience, including dose reduction

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Effect of miglustat on bone disease in adults with type 1 Gaucher disease: a pooled analysis of three multinational, open-label studies.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Oral maintenance clinical trial with miglustat for type I Gaucher disease: switch from or combination with intravenous enzyme replacement.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Miglustat (Zavesca) in type 1 Gaucher disease: 5-year results of a post-authorisation safety surveillance programme.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Dietary modifications in patients receiving miglustat.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

Skeletal complications of Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.08.004

inferred from DOI database lookup

7 January 2021

based on heuristic

Identifiers

DOI

10.1016/J.YMGME.2011.08.004

1 reference

25 August 2017

1 reference

PubMed ID

21889384