Eight years experience with enzyme replacement therapy in two children and one adult with Pompe disease.
about
Hearing loss in Pompe disease revisited: results from a study of 24 children.Fatigue in neuromuscular disorders: focus on Guillain-Barré syndrome and Pompe diseaseHypertrophic cardiomyopathy in pompe disease is not limited to the classic infantile-onset phenotype.Consensus treatment recommendations for late-onset Pompe diseaseEnzyme replacement therapy in late-onset Pompe disease: a systematic literature review.Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy.Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study.Lysosomal Storage Diseases-Regulating Neurodegeneration.Neural deficits contribute to respiratory insufficiency in Pompe disease.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.Glycoengineered acid alpha-glucosidase with improved efficacy at correcting the metabolic aberrations and motor function deficits in a mouse model of Pompe disease.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.Evaluation of systemic follistatin as an adjuvant to stimulate muscle repair and improve motor function in Pompe mice.36 months observational clinical study of 38 adult Pompe disease patients under alglucosidase alfa enzyme replacement therapy.Abnormal mannose-6-phosphate receptor trafficking impairs recombinant alpha-glucosidase uptake in Pompe disease fibroblasts.Enzyme replacement in neuronal storage disorders in the pediatric population.Muscle fiber-type distribution, fiber-type-specific damage, and the Pompe disease phenotype.Low-frequency enzyme replacement therapy in late-onset Pompe disease.The humanistic burden of Pompe disease: are there still unmet needs? A systematic review.A molecular analysis of the GAA gene and clinical spectrum in 38 patients with Pompe disease in Japan.Observational clinical study in juvenile-adult glycogenosis type 2 patients undergoing enzyme replacement therapy for up to 4 years.Enzyme replacement therapy improves respiratory outcomes in patients with late-onset type II glycogenosis and high ventilator dependency.Fibromyalgia-like symptoms associated with irritable bowel syndrome: A challenging diagnosis of late-onset Pompe disease.Effectiveness of enzyme replacement therapy in adults with late-onset Pompe disease: results from the NCS-LSD cohort study.Effects of enzyme replacement therapy on five patients with advanced late-onset glycogen storage disease type II: a 2-year follow-up study
P2860
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P2860
Eight years experience with enzyme replacement therapy in two children and one adult with Pompe disease.
description
2008 nî lūn-bûn
@nan
2008年の論文
@ja
2008年論文
@yue
2008年論文
@zh-hant
2008年論文
@zh-hk
2008年論文
@zh-mo
2008年論文
@zh-tw
2008年论文
@wuu
2008年论文
@zh
2008年论文
@zh-cn
name
Eight years experience with en ...... one adult with Pompe disease.
@en
type
label
Eight years experience with en ...... one adult with Pompe disease.
@en
prefLabel
Eight years experience with en ...... one adult with Pompe disease.
@en
P2093
P1476
Eight years experience with en ...... one adult with Pompe disease.
@en
P2093
A J J Reuser
A T van der Ploeg
C I van Capelle
L P F Winkel
M L C Hagemans
P A van Doorn
S K Shapira
W F M Arts
P304
P356
10.1016/J.NMD.2008.04.009
P577
2008-05-27T00:00:00Z