Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational study
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The clinical relevance of outcomes used in late-onset Pompe disease: can we do better?Pathophysiological concepts in the congenital myopathies: blurring the boundaries, sharpening the focusSkeletal muscle quantitative nuclear magnetic resonance imaging follow-up of adult Pompe patientsIncreased aortic stiffness and blood pressure in non-classic Pompe disease.Late-Onset Glycogen Storage Disease Type II (Pompe's Disease) with a Novel Mutation: A Malaysian Experience.Urge incontinence and gastrointestinal symptoms in adult patients with pompe disease: a cross-sectional surveyPompe disease: literature review and case seriesDiagnosis and treatment of late-onset Pompe disease in the Middle East and North Africa region: consensus recommendations from an expert groupSafety 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.Familial adult-onset Pompe disease associated with unusual clinical and histological features.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.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.Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease.Screening for late-onset Pompe disease in western Denmark.Respiratory manifestations in late-onset Pompe disease: a case series conducted in Brazil.Effectiveness of enzyme replacement therapy in adults with late-onset Pompe disease: results from the NCS-LSD cohort study.Identification and characterization of aberrant GAA pre-mRNA splicing in pompe disease using a generic approach.Orthopedic management of patients with Pompe disease: a retrospective case series of 8 patients
P2860
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P2860
Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational study
description
2013 nî lūn-bûn
@nan
2013年の論文
@ja
2013年論文
@yue
2013年論文
@zh-hant
2013年論文
@zh-hk
2013年論文
@zh-mo
2013年論文
@zh-tw
2013年论文
@wuu
2013年论文
@zh
2013年论文
@zh-cn
name
Impact of enzyme replacement t ...... ernational observational study
@ast
Impact of enzyme replacement t ...... ernational observational study
@en
type
label
Impact of enzyme replacement t ...... ernational observational study
@ast
Impact of enzyme replacement t ...... ernational observational study
@en
prefLabel
Impact of enzyme replacement t ...... ernational observational study
@ast
Impact of enzyme replacement t ...... ernational observational study
@en
P2093
P2860
P356
P1476
Impact of enzyme replacement t ...... ernational observational study
@en
P2093
Ans T van der Ploeg
Arnold J J Reuser
Deniz Güngör
Marloes L C Hagemans
Michelle E Kruijshaar
Ralph B D'Agostino
P2860
P2888
P356
10.1186/1750-1172-8-49
P577
2013-03-27T00:00:00Z
P5875
P6179
1040028754