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注意:仅列出部分参考文献,下载原文获取全部文献信息。Facial-muscle weakness, speech disorders and dysphagia are common in patients with classic infantile Pompe disease treated with enzyme therapy
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JOURNAL OF INHERITED METABOLIC DISEASE (2012)
36 months observational clinical study of 38 adult Pompe disease patients under alglucosidase alfa enzyme replacement therapy
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Observational clinical study in juvenile-adult glycogenosis type 2 patients undergoing enzyme replacement therapy for up to 4 years
C. Angelini et al.
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Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study
Juna M. de Vries et al.
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Recombinant human acid alpha-glucosidase (rhGAA) in adult patients with severe respiratory failure due to Pompe disease
David Orlikowski et al.
NEUROMUSCULAR DISORDERS (2011)
Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy
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ORPHANET JOURNAL OF RARE DISEASES (2011)
Long-term observational, non-randomized study of enzyme replacement therapy in late-onset glycogenosis type II
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JOURNAL OF INHERITED METABOLIC DISEASE (2010)
Enzyme replacement therapy with alglucosidase alfa in 44 patients with late-onset glycogen storage disease type 2: 12-month results of an observational clinical trial
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JOURNAL OF NEUROLOGY (2010)
A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease.
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NEW ENGLAND JOURNAL OF MEDICINE (2010)
Early Treatment With Alglucosidase Alfa Prolongs Long-Term Survival of Infants With Pompe Disease
Priya S. Kishnani et al.
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Lysosomal storage disease 2 - Pompe's disease
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Immortal time bias in observational studies of drug effects
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JOURNAL OF PEDIATRICS (2006)
Disease severity in children and adults with Pompe disease related to age and disease duration
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NEUROLOGY (2005)
Clinical manifestation and natural course of late-onset Pompe's disease in 54 Dutch patients
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BRAIN (2005)
Late-onset Pompe disease primarily affects quality of life in physical health domains
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NEUROLOGY (2004)
Enzyme therapy for Pompe disease with recombinant human α-glucosidase from rabbit milk
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Recombinant human α-glucosidase from rabbit milk in Pompe patients
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LANCET (2000)
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NEW ENGLAND JOURNAL OF MEDICINE (2000)