The Gaucher registry: demographics and disease characteristics of 1698 patients with Gaucher disease.
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Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type IEnzyme replacement and substrate reduction therapy for Gaucher diseaseEnzyme replacement therapy with laronidase (Aldurazyme) for treating mucopolysaccharidosis type IEnzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type IEnzyme replacement therapy with laronidase (Aldurazyme ® ) for treating mucopolysaccharidosis type ILife expectancy in Gaucher disease type 1Type I Gaucher disease with exophthalmos and pulmonary arteriovenous malformationClinical manifestations and management of Gaucher diseaseThe role of saposin C in Gaucher diseaseUnderstanding the natural history of Gaucher diseaseX-ray structure of human acid-β-glucosidase, the defective enzyme in Gaucher diseaseAcid beta-glucosidase: insights from structural analysis and relevance to Gaucher disease therapyDecreased glucocerebrosidase activity in Gaucher disease parallels quantitative enzyme loss due to abnormal interaction with TCP1 and c-Cbl.Imiglucerase in the management of Gaucher disease type 1: an evidence-based review of its place in therapyImiglucerase and its use for the treatment of Gaucher's diseaseIn vivo and ex vivo evaluation of L-type calcium channel blockers on acid beta-glucosidase in Gaucher disease mouse modelsHigh throughput screening for small molecule therapy for Gaucher disease using patient tissue as the source of mutant glucocerebrosidaseGaucher disease: transcriptome analyses using microarray or mRNA sequencing in a Gba1 mutant mouse model treated with velaglucerase alfa or imigluceraseA Review of Gaucher Disease Pathophysiology, Clinical Presentation and TreatmentsRandomized, controlled trial of miglustat in Gaucher's disease type 3.Velaglucerase alfa (VPRIV) enzyme replacement therapy in patients with Gaucher disease: Long-term data from phase III clinical trials.Individualization of long-term enzyme replacement therapy for Gaucher disease.Mapping the genetic and clinical characteristics of Gaucher disease in the Iberian Peninsula.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.Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatmentThe French Gaucher's disease registry: clinical characteristics, complications and treatment of 562 patients.A case of adult type 1 Gaucher disease complicated by temporal intestinal hemorrhage.Gaucher disease in a family from MaranhãoHematological manifestations and complications of Gaucher disease.Rare Case of Hepatic Gaucheroma in a Child on Enzyme Replacement Therapy.Treatment of profound thrombocytopenia in a patient with Gaucher disease type 1: Is there a role for substrate reduction therapy.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).The incidence of Parkinsonism in patients with type 1 Gaucher disease: data from the ICGG Gaucher RegistryThe European Gaucher Alliance: a survey of member patient organisations' activities, healthcare environments and concerns.Metabolic cardiomyopathies.ELIGLUSTAT TARTRATE: Glucosylceramide Synthase Inhibitor Treatment of Type 1 Gaucher Disease.Hepatobiliary quiz-10 (2014)The underrecognized progressive nature of N370S Gaucher disease and assessment of cancer risk in 403 patients
P2860
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P2860
The Gaucher registry: demographics and disease characteristics of 1698 patients with Gaucher disease.
description
2000 nî lūn-bûn
@nan
2000 թուականի Հոկտեմբերին հրատարակուած գիտական յօդուած
@hyw
2000 թվականի հոտեմբերին հրատարակված գիտական հոդված
@hy
2000年の論文
@ja
2000年論文
@yue
2000年論文
@zh-hant
2000年論文
@zh-hk
2000年論文
@zh-mo
2000年論文
@zh-tw
2000年论文
@wuu
name
The Gaucher registry: demograp ...... patients with Gaucher disease.
@ast
The Gaucher registry: demograp ...... patients with Gaucher disease.
@en
The Gaucher registry: demograp ...... patients with Gaucher disease.
@nl
type
label
The Gaucher registry: demograp ...... patients with Gaucher disease.
@ast
The Gaucher registry: demograp ...... patients with Gaucher disease.
@en
The Gaucher registry: demograp ...... patients with Gaucher disease.
@nl
prefLabel
The Gaucher registry: demograp ...... patients with Gaucher disease.
@ast
The Gaucher registry: demograp ...... patients with Gaucher disease.
@en
The Gaucher registry: demograp ...... patients with Gaucher disease.
@nl
P2093
P1476
The Gaucher registry: demograp ...... patients with Gaucher disease.
@en
P2093
Andersson HC
Kolodny EH
Pastores G
Rosenbloom BE
Wappner RS
Weinreb NJ
P304
P356
10.1001/ARCHINTE.160.18.2835
P407
P577
2000-10-01T00:00:00Z