Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study.
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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 MRIMuscle imaging data in late-onset Pompe disease reveal a correlation between the pre-existing degree of lipomatous muscle alterations and the efficacy of long-term enzyme replacement therapy.GAA Deficiency in Pompe Disease Is Alleviated by Exon Inclusion in iPSC-Derived Skeletal Muscle Cells.Urge incontinence and gastrointestinal symptoms in adult patients with pompe disease: a cross-sectional surveyLung MRI and impairment of diaphragmatic function in Pompe disease.Safety and efficacy of exercise training in adults with Pompe disease: evalution of endurance, muscle strength and core stability before and after a 12 week training programA conceptual disease model for adult Pompe disease.Quality of life and participation in daily life of adults with Pompe disease receiving enzyme replacement therapy: 10 years of international follow-up.Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational studyTen 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.Survival and long-term outcomes in late-onset Pompe disease following alglucosidase alfa treatment: a systematic review and meta-analysis.European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10-year experience.Clinical and molecular aspects of 30 patients with late-onset Pompe disease (LOPD): unusual features and response to treatment.AAV-mediated transcription factor EB (TFEB) gene delivery ameliorates muscle pathology and function in the murine model of Pompe Disease.Pompe disease in Austria: clinical, genetic and epidemiological aspects.Respiratory manifestations in late-onset Pompe disease: a case series conducted in Brazil.PRKAG2 mutations presenting in infancy.Comparison of EQ-5D and SF-6D utilities in Pompe disease.Pompe disease in adulthood: effects of antibody formation on enzyme replacement therapy.Cessation and resuming of alglucosidase alfa in Pompe disease: a retrospective analysis.Enzyme replacement therapy reduces the risk for wheelchair dependency in adult Pompe patients.
P2860
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P2860
Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study.
description
2012 nî lūn-bûn
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2012年の論文
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2012年学术文章
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2012年学术文章
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2012年学术文章
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2012年学术文章
@zh-my
2012年学术文章
@zh-sg
2012年學術文章
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name
Effect of enzyme therapy and p ...... pen-label single-center study.
@ast
Effect of enzyme therapy and p ...... pen-label single-center study.
@en
type
label
Effect of enzyme therapy and p ...... pen-label single-center study.
@ast
Effect of enzyme therapy and p ...... pen-label single-center study.
@en
prefLabel
Effect of enzyme therapy and p ...... pen-label single-center study.
@ast
Effect of enzyme therapy and p ...... pen-label single-center study.
@en
P2093
P2860
P356
P1476
Effect of enzyme therapy and p ...... pen-label single-center study.
@en
P2093
Anneke J van der Kooi
Ans T van der Ploeg
Arnold J J Reuser
Baziel G M van Engelen
Catharina G Faber
Francois P J Karstens
Jan B M Kuks
Jan J G M Verschuuren
John H Wokke
Juna M de Vries
P2860
P2888
P356
10.1186/1750-1172-7-73
P577
2012-09-26T00:00:00Z
P5875
P6179
1013668291