Recommendations for the use of eliglustat in the treatment of adults with Gaucher disease type 1 in the United States (Q26783092)

1

0 references

Thomas Andrew Burrow

2

0 references

Joel Charrow

3

0 references

Ozlem Goker-Alpan

4

0 references

Paige Kaplan

5

0 references

Pramod Mistry

7

0 references

Jeremy Ruskin

8

0 references

Neal Weinreb

Molecular Genetics and Metabolism

9

0 references

language of work or name

English

0 references

publication date

February 2016

0 references

published in

0 references

volume

117

0 references

issue

2

0 references

page(s)

95-103

0 references

cites work

Pediatric non-neuronopathic Gaucher disease: presentation, diagnosis and assessment. Consensus statements

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

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.2015.09.002

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

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.2015.09.002

Pulmonary hypertension in type 1 Gaucher's disease: genetic and epigenetic determinants of phenotype and response to therapy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

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.2015.09.002

Therapeutic goals in the treatment of Gaucher disease.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

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.2015.09.002

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

Quality of life related to type 1 Gaucher disease: Spanish experience

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

The impact of Gaucher disease and its treatment on quality of life

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

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.2015.09.002

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.2015.09.002

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.2015.09.002

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.2015.09.002

Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

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.2015.09.002

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.2015.09.002

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.2015.09.002

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.2015.09.002

A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

Clinical Pharmacogenetics Implementation Consortium guideline for CYP2D6 and CYP2C19 genotypes and dosing of tricyclic antidepressants

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

Skeletal improvement in patients with Gaucher disease type 1: a phase 2 trial of oral eliglustat

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

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.2015.09.002

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.2015.09.002

Eliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2 trial results after 4 years of treatment

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

Eliglustat compared with imiglucerase in patients with Gaucher's disease type 1 stabilised on enzyme replacement therapy: a phase 3, randomised, open-label, non-inferiority trial.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

Gaucher disease: recommendations on diagnosis, evaluation, and monitoring

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

A reappraisal of Gaucher disease-diagnosis and disease management algorithms.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

Gaucher disease type 1: revised recommendations on evaluations and monitoring for adult patients

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

Outcome of pregnancies in women receiving velaglucerase alfa for Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

The management of pregnancy in Gaucher disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

Successful pregnancy and lactation outcome in a patient with Gaucher disease receiving enzyme replacement therapy, and the subsequent distribution and excretion of imiglucerase in human breast milk.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

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.2015.09.002

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.2015.09.002

Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

Risk factors for fractures and avascular osteonecrosis in type 1 Gaucher disease: a study from the International Collaborative Gaucher Group (ICGG) Gaucher Registry.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

Evaluation of disease burden and response to treatment in adults with type 1 Gaucher disease using a validated disease severity scoring system (DS3)

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2015.09.002

10.1016/J.YMGME.2015.09.002

7 January 2021

Identifiers

DOI

1 reference

Consolidated OpenCitations Corpus – April 2017

OpenCitations bibliographic resource ID

1990267

Fatcat ID

release_ceakvpsl3vfj7nferc37ws6xcq

0 references

OpenCitations bibliographic resource ID

1990267

1 reference