A benchmark analysis of the achievement of therapeutic goals for type 1 Gaucher disease patients treated with imiglucerase.
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Imiglucerase 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 therapyGenome-wide association study of N370S homozygous Gaucher disease reveals the candidacy of CLN8 gene as a genetic modifier contributing to extreme phenotypic variationCharacteristics of type I Gaucher disease associated with persistent thrombocytopenia after treatment with imiglucerase for 4-5 years.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.Achievement of therapeutic goals with low-dose imiglucerase in Gaucher disease: a single-center experience.Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy.[International Children's Day]The underrecognized progressive nature of N370S Gaucher disease and assessment of cancer risk in 403 patientsGaucher disease: clinical profile and therapeutic developments.Patients with type 1 Gaucher disease in South Florida, USA: demographics, genotypes, disease severity and treatment outcomes.Misdiagnosis of Niemann-Pick disease type C as Gaucher diseaseA 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.Evaluation of high density lipoprotein as a circulating biomarker of Gaucher disease activityReducing 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 1Evaluation of disease burden and response to treatment in adults with type 1 Gaucher disease using a validated disease severity scoring system (DS3)Time of Initiating Enzyme Replacement Therapy Affects Immune Abnormalities and Disease Severity in Patients with Gaucher Disease.Safety and efficacy of velaglucerase alfa in Gaucher disease type 1 patients previously treated with imiglucerase.Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapyReview of the safety and efficacy of imiglucerase treatment of Gaucher disease.Real-world clinical experience with long-term miglustat maintenance therapy in type 1 Gaucher disease: the ZAGAL project.Glucocerebroside: an evolutionary advantage for patients with Gaucher disease and a new immunomodulatory agent.Enzyme therapy for the treatment of type 1 Gaucher disease: clinical outcomes and dose - response relationships.A new framework for evaluating the health impacts of treatment for Gaucher disease type 1.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 1Long-term treatment outcomes in Gaucher disease.Skeletal involvement in Gaucher disease: An observational multicenter study of prognostic factors in the Argentine Gaucher disease patients.The history and accomplishments of the ICGG Gaucher registry.A Novel Nonsense Mutation of the AGL Gene in a Romanian Patient with Glycogen Storage Disease Type IIIa.
P2860
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P2860
A benchmark analysis of the achievement of therapeutic goals for type 1 Gaucher disease patients treated with imiglucerase.
description
2008 nî lūn-bûn
@nan
2008年の論文
@ja
2008年学术文章
@wuu
2008年学术文章
@zh
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
A benchmark analysis of the ac ...... nts treated with imiglucerase.
@en
A benchmark analysis of the ac ...... nts treated with imiglucerase.
@nl
type
label
A benchmark analysis of the ac ...... nts treated with imiglucerase.
@en
A benchmark analysis of the ac ...... nts treated with imiglucerase.
@nl
prefLabel
A benchmark analysis of the ac ...... nts treated with imiglucerase.
@en
A benchmark analysis of the ac ...... nts treated with imiglucerase.
@nl
P2093
P356
P1476
A benchmark analysis of the ac ...... nts treated with imiglucerase.
@en
P2093
John Taylor
Neal Weinreb
Stephan vom Dahl
Timothy Cox
P2860
P304
P356
10.1002/AJH.21280
P577
2008-12-01T00:00:00Z