Eight-year clinical outcomes of long-term enzyme replacement therapy for 884 children with Gaucher disease type 1.
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Enzyme replacement and substrate reduction therapy for Gaucher diseaseImiglucerase in the treatment of Gaucher disease: a history and perspectiveImiglucerase in the management of Gaucher disease type 1: an evidence-based review of its place in therapyVelaglucerase alfa (VPRIV) enzyme replacement therapy in patients with Gaucher disease: Long-term data from phase III clinical trials.An overview on bone manifestations in Gaucher disease.Coxarthritis as the presenting symptom of Gaucher disease type 1.Characteristics of type I Gaucher disease associated with persistent thrombocytopenia after treatment with imiglucerase for 4-5 years.Revised recommendations for the management of Gaucher disease in children.Achievement of therapeutic goals with low-dose imiglucerase in Gaucher disease: a single-center experience.Diagnosis features of pediatric Gaucher disease patients in the era of enzymatic therapy, a national-base study from the Spanish Registry of Gaucher Disease.Vertebral fractures in Gaucher disease type I: data from the French "Observatoire" on Gaucher disease (FROG).Imaging of Gaucher disease.Gaucher disease: clinical profile and therapeutic developments.Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders.Study of enzyme replacement therapy for Gaucher Disease: comparative analysis of clinical and laboratory parameters at diagnosis and after two, five and ten years of treatment.Reducing selection bias in case-control studies from rare disease registries.Switching from imiglucerase to miglustat for the treatment of French patients with Gaucher disease type 1: a case series.Bone turnover markers in patients with type 1 Gaucher disease.Review of the safety and efficacy of imiglucerase treatment of Gaucher disease.Enzyme therapy for the treatment of type 1 Gaucher disease: clinical outcomes and dose - response relationships.Gauchers disease--a reappraisal of hematopoietic stem cell transplantation.Effects of imiglucerase on the growth and metabolism of Gaucher disease type I patients: a systematic review.Early diagnosis of Gaucher disease in pediatric patients: proposal for a diagnostic algorithm.Gaucher disease and bone manifestations.Management of bone disease in Gaucher disease type 1: clinical practice.Velaglucerase alfa in the treatment of Gaucher disease type 1Pathogenesis of Bone Alterations in Gaucher Disease: The Role of Immune System.Gaucher disease types 1 and 3: Phenotypic characterization of large populations from the ICGG Gaucher Registry.Long-term treatment outcomes in Gaucher disease.Skeletal involvement in Gaucher disease: An observational multicenter study of prognostic factors in the Argentine Gaucher disease patients.Gaucher disease in Iraqi children (Clinical, diagnostic & therapeutic aspects).Gaucher disease in Syrian children: common mutations identification, and clinical futures.58th annual meeting american society of human genetics, 2008The history and accomplishments of the ICGG Gaucher registry.Transformation in pretreatment manifestations of Gaucher disease type 1 during two decades of alglucerase/imiglucerase enzyme replacement therapy in the International Collaborative Gaucher Group (ICGG) Gaucher Registry.Optimal therapy in Gaucher diseaseEnzyme replacement in neuronal storage disorders in the pediatric population.Long-term effectiveness of enzyme replacement therapy in adults with Gaucher disease: results from the NCS-LSD cohort study.Dynamic changes of lipid profile in Romanian patients with Gaucher disease type 1 under enzyme replacement therapy: a prospective study.Plasma oligomeric alpha-synuclein is associated with glucocerebrosidase activity in Gaucher disease.
P2860
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P2860
Eight-year clinical outcomes of long-term enzyme replacement therapy for 884 children with Gaucher disease type 1.
description
2008 nî lūn-bûn
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2008年の論文
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2008年学术文章
@wuu
2008年学术文章
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2008年学术文章
@zh-cn
2008年学术文章
@zh-hans
2008年学术文章
@zh-my
2008年学术文章
@zh-sg
2008年學術文章
@yue
2008年學術文章
@zh-hant
name
Eight-year clinical outcomes o ...... n with Gaucher disease type 1.
@en
Eight-year clinical outcomes o ...... n with Gaucher disease type 1.
@nl
type
label
Eight-year clinical outcomes o ...... n with Gaucher disease type 1.
@en
Eight-year clinical outcomes o ...... n with Gaucher disease type 1.
@nl
prefLabel
Eight-year clinical outcomes o ...... n with Gaucher disease type 1.
@en
Eight-year clinical outcomes o ...... n with Gaucher disease type 1.
@nl
P2093
P356
P1433
P1476
Eight-year clinical outcomes o ...... n with Gaucher disease type 1.
@en
P2093
Hans Andersson
Katherine Kacena
Paige Kaplan
P304
P356
10.1542/PEDS.2007-2144
P407
P577
2008-12-01T00:00:00Z