Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy.
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The clinical relevance of outcomes used in late-onset Pompe disease: can we do better?Quantification of Diaphragm Mechanics in Pompe Disease Using Dynamic 3D MRIRespiratory function in late-onset Pompe disease patients receiving long-term enzyme replacement therapy for more than 48 months.Lung MRI and impairment of diaphragmatic function in Pompe disease.A conceptual disease model for adult Pompe disease.Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational studyPrevalence of Pompe disease in 3,076 patients with hyperCKemia and limb-girdle muscular weakness.Therapeutic advances in the management of Pompe disease and other metabolic myopathiesMaximum inspiratory pressure as a clinically meaningful trial endpoint for neuromuscular diseases: a comprehensive review of the literature.Enzyme replacement therapy for Pompe disease.Ten years of the international Pompe survey: patient reported outcomes as a reliable tool for studying treated and untreated children and adults with non-classic Pompe disease.Guidelines for the diagnosis, treatment and clinical monitoring of patients with juvenile and adult Pompe disease.Pompe Disease: Diagnosis and Management. Evidence-Based Guidelines from a Canadian Expert Panel.The role of immune tolerance induction in restoration of the efficacy of ERT in Pompe disease.Toward deconstructing the phenotype of late-onset Pompe disease.The Outcome of Infantile Onset Pompe Disease in South of Iran.Enzyme enhancers for the treatment of Fabry and Pompe disease.Early is better? A new algorithm for early diagnosis in late onset Pompe disease (LOPD).Update of the pompe disease mutation database with 60 novel GAA sequence variants and additional studies on the functional effect of 34 previously reported variants.Pompe disease, the must-not-miss diagnosis: A report of 3 patients.S2.2 Enzyme replacement therapyin the infantile-onset Pompe disease.S1.4 Cardiovascular involvement in Pompedisease.Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease.Late-onset Pompe's disease in a hemodialysis patient: A first case report.Respiratory manifestations in late-onset Pompe disease: a case series conducted in Brazil.Sleep-related symptoms and sleep-disordered breathing in adult Pompe disease.Comparison of EQ-5D and SF-6D utilities in Pompe disease.Swiss national guideline for reimbursement of enzyme replacement therapy in late-onset Pompe disease.Effectiveness of enzyme replacement therapy in adults with late-onset Pompe disease: results from the NCS-LSD cohort study.
P2860
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P2860
Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy.
description
2011 nî lūn-bûn
@nan
2011 թուականի Յունիսին հրատարակուած գիտական յօդուած
@hyw
2011 թվականի հունիսին հրատարակված գիտական հոդված
@hy
2011年の論文
@ja
2011年論文
@yue
2011年論文
@zh-hant
2011年論文
@zh-hk
2011年論文
@zh-mo
2011年論文
@zh-tw
2011年论文
@wuu
name
Survival and associated factor ...... th enzyme replacement therapy.
@ast
Survival and associated factor ...... th enzyme replacement therapy.
@en
type
label
Survival and associated factor ...... th enzyme replacement therapy.
@ast
Survival and associated factor ...... th enzyme replacement therapy.
@en
prefLabel
Survival and associated factor ...... th enzyme replacement therapy.
@ast
Survival and associated factor ...... th enzyme replacement therapy.
@en
P2093
P2860
P356
P1476
Survival and associated factor ...... th enzyme replacement therapy.
@en
P2093
Ans T van der Ploeg
Arnold J J Reuser
Deniz Güngör
Juna M de Vries
Marloes L C Hagemans
Wim C J Hop
P2860
P2888
P356
10.1186/1750-1172-6-34
P577
2011-06-01T00:00:00Z
P5875
P6179
1039402261