The clinical and demographic characteristics of nonneuronopathic Gaucher disease in 887 children at diagnosis.
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Enzyme replacement and substrate reduction therapy for Gaucher diseaseEnzyme replacement and substrate reduction therapy for Gaucher diseaseImiglucerase in the treatment of Gaucher disease: a history and perspectiveUnderstanding the natural history of Gaucher diseaseImiglucerase and its use for the treatment of Gaucher's diseaseA Review of Gaucher Disease Pathophysiology, Clinical Presentation and TreatmentsNovel mutations in the glucocerebrosidase gene of Indian patients with Gaucher disease.Biomarkers for disease progression and AAV therapeutic efficacy in feline Sandhoff diseaseType 1 Gaucher disease: significant disease manifestations in "asymptomatic" homozygotes.Coxarthritis as the presenting symptom of Gaucher disease type 1.Splenomegaly, cardiomegaly, and osteoporosis in a child with Gaucher disease.Revised recommendations for the management of Gaucher disease in children.Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatmentGaucher disease in a family from MaranhãoHematological manifestations and complications of Gaucher disease.Rare Case of Hepatic Gaucheroma in a Child on Enzyme Replacement Therapy.Vertebral fractures in Gaucher disease type I: data from the French "Observatoire" on Gaucher disease (FROG).Modeling changes in biomarkers in Gaucher disease patients receiving enzyme replacement therapy using a pathophysiological model.The European Gaucher Alliance: a survey of member patient organisations' activities, healthcare environments and concerns.ELIGLUSTAT TARTRATE: Glucosylceramide Synthase Inhibitor Treatment of Type 1 Gaucher Disease.The genetics of Parkinson disease.A reappraisal of Gaucher disease-diagnosis and disease management algorithms.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.Impact of imiglucerase supply constraint on the therapeutic management and course of disease in French patients with Gaucher disease type 1Recommendations for the management of the haematological and onco-haematological aspects of Gaucher disease.Successful screening for Gaucher disease in a high-prevalence population in tabuleiro do Norte (northeastern Brazil): a cross-sectional studyReview of the safety and efficacy of imiglucerase treatment of Gaucher disease.Timing of initiation of enzyme replacement therapy after diagnosis of type 1 Gaucher disease: effect on incidence of avascular necrosis.Glucocerebrosidase involvement in Parkinson disease and other synucleinopathies.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: the metabolic defect, pathophysiology, phenotypes and natural history.Management of bone disease in Gaucher disease type 1: clinical practice.Gaucher disease types 1 and 3: Phenotypic characterization of large populations from the ICGG Gaucher Registry.Lysosomal storage disorders: A review of the musculoskeletal features.Early manifestations of type 1 Gaucher disease in presymptomatic children diagnosed after parental carrier screening.A disease severity scoring system for children with type 1 Gaucher disease.The 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.
P2860
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P2860
The clinical and demographic characteristics of nonneuronopathic Gaucher disease in 887 children at diagnosis.
description
2006 nî lūn-bûn
@nan
2006 թուականի Յունիսին հրատարակուած գիտական յօդուած
@hyw
2006 թվականի հունիսին հրատարակված գիտական հոդված
@hy
2006年の論文
@ja
2006年論文
@yue
2006年論文
@zh-hant
2006年論文
@zh-hk
2006年論文
@zh-mo
2006年論文
@zh-tw
2006年论文
@wuu
name
The clinical and demographic c ...... in 887 children at diagnosis.
@ast
The clinical and demographic c ...... in 887 children at diagnosis.
@en
type
label
The clinical and demographic c ...... in 887 children at diagnosis.
@ast
The clinical and demographic c ...... in 887 children at diagnosis.
@en
prefLabel
The clinical and demographic c ...... in 887 children at diagnosis.
@ast
The clinical and demographic c ...... in 887 children at diagnosis.
@en
P2093
P1433
P1476
The clinical and demographic c ...... in 887 children at diagnosis.
@en
P2093
Hans C Andersson
John D Yee
Katherine A Kacena
Paige Kaplan
P304
P356
10.1001/ARCHPEDI.160.6.603
P407
P577
2006-06-01T00:00:00Z