Facial-muscle weakness, speech disorders and dysphagia are common in patients with classic infantile Pompe disease treated with enzyme therapy
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Knockout of Lysosomal Enzyme-Targeting Gene Causes Abnormalities in Mouse Pup Isolation CallsThe emerging phenotype of long-term survivors with infantile Pompe disease.Swallow Prognosis and Follow-Up Protocol in Infantile Onset Pompe DiseaseHard to swallow: Developmental biological insights into pediatric dysphagia.Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational studyEffects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center studyPhase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes.Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy.Lower Urinary Tract Symptoms and Incontinence in Children with Pompe DiseaseFamilial adult-onset Pompe disease associated with unusual clinical and histological features.Feeding and Swallowing Disorders in Pediatric Neuromuscular Diseases: An Overview.Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice.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.Pompe Disease: Diagnosis and Management. Evidence-Based Guidelines from a Canadian Expert Panel.Screening and evaluation tools of dysphagia in children with neuromuscular diseases: a systematic review.A Neuron-Specific Gene Therapy Relieves Motor Deficits in Pompe Disease Mice.Improvement of dysphagia in a child affected by Pompe disease treated with enzyme replacement therapy.Long-term neurologic and cardiac correction by intrathecal gene therapy in Pompe disease.Enzyme replacement in neuronal storage disorders in the pediatric population.The humanistic burden of Pompe disease: are there still unmet needs? A systematic review.Follow-up analysis of voice quality in patients with late-onset Pompe disease
P2860
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P2860
Facial-muscle weakness, speech disorders and dysphagia are common in patients with classic infantile Pompe disease treated with enzyme therapy
description
2011 nî lūn-bûn
@nan
2011年の論文
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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
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2011年论文
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2011年论文
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name
Facial-muscle weakness, speech ...... se treated with enzyme therapy
@ast
Facial-muscle weakness, speech ...... se treated with enzyme therapy
@en
type
label
Facial-muscle weakness, speech ...... se treated with enzyme therapy
@ast
Facial-muscle weakness, speech ...... se treated with enzyme therapy
@en
prefLabel
Facial-muscle weakness, speech ...... se treated with enzyme therapy
@ast
Facial-muscle weakness, speech ...... se treated with enzyme therapy
@en
P2093
P2860
P1476
Facial-muscle weakness, speech ...... se treated with enzyme therapy
@en
P2093
A J J Reuser
A T van der Ploeg
B J Ebbink
C I van Capelle
C M van Gelder
H H W de Gier
I F M de Coo
I Moor-van Nugteren
J M P van den Hout
M M Hakkesteegt
P2860
P2888
P304
P356
10.1007/S10545-011-9404-7
P577
2011-10-19T00:00:00Z