Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease.
about
Clinical features and predictors for disease natural progression in adults with Pompe disease: a nationwide prospective observational studyEnzyme replacement therapy for infantile-onset Pompe diseaseTargeted approaches to induce immune tolerance for Pompe disease therapyNew insights into therapeutic options for Pompe diseaseThe pharmacological chaperone AT2220 increases the specific activity and lysosomal delivery of mutant acid alpha-glucosidase, and promotes glycogen reduction in a transgenic mouse model of Pompe diseaseDurable and sustained immune tolerance to ERT in Pompe disease with entrenched immune responsesHearing in adults with Pompe diseaseUse of cardiac magnetic resonance imaging to evaluate cardiac structure, function and fibrosis in children with infantile Pompe disease on enzyme replacement therapy.The emerging phenotype of long-term survivors with infantile Pompe disease.N-glycans of recombinant human acid alpha-glucosidase expressed in the milk of transgenic rabbits.Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease.Respiratory muscle training with enzyme replacement therapy improves muscle strength in late - onset Pompe disease.Immunomodulatory gene therapy in lysosomal storage disordersAutophagy in skeletal muscle: implications for Pompe diseaseRole of autophagy in the pathogenesis of Pompe disease.The pharmacological chaperone AT2220 increases recombinant human acid α-glucosidase uptake and glycogen reduction in a mouse model of Pompe disease.Differences in the predominance of lysosomal and autophagic pathologies between infants and adults with Pompe disease: implications for therapy.Pompe disease diagnosis and management guideline.Cardiovascular abnormalities in late-onset Pompe disease and response to enzyme replacement therapyThe impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease.Consensus treatment recommendations for late-onset Pompe diseaseEnzyme replacement therapy in late-onset Pompe disease: a systematic literature review.Algorithm for the early diagnosis and treatment of patients with cross reactive immunologic material-negative classic infantile pompe disease: a step towards improving the efficacy of ERT.Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe diseaseSurvival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy.Enzyme therapy and immune response in relation to CRIM status: the Dutch experience in classic infantile Pompe disease.Assessment of toxicity and biodistribution of recombinant AAV8 vector-mediated immunomodulatory gene therapy in mice with Pompe disease.Outcome of patients with classical infantile pompe disease receiving enzyme replacement therapy in Germany.Predicting cross-reactive immunological material (CRIM) status in Pompe disease using GAA mutations: lessons learned from 10 years of clinical laboratory testing experience.Toward reducing immunogenicity of enzyme replacement therapy: altering the specificity of human β-glucuronidase to compensate for α-iduronidase deficiency.Postmortem Findings and Clinical Correlates in Individuals with Infantile-Onset Pompe DiseaseDiagnosis and treatment of late-onset Pompe disease in the Middle East and North Africa region: consensus recommendations from an expert groupImmune Tolerance Strategies in Siblings with Infantile Pompe Disease-Advantages for a Preemptive Approach to High-Sustained Antibody Titers.Facial-muscle weakness, speech disorders and dysphagia are common in patients with classic infantile Pompe disease treated with enzyme therapyCorrecting Neuromuscular Deficits With Gene Therapy in Pompe DiseaseCRIM-negative infantile Pompe disease: characterization of immune responses in patients treated with ERT monotherapyLack of robust satellite cell activation and muscle regeneration during the progression of Pompe disease.Clinical Laboratory Experience of Blood CRIM Testing in Infantile Pompe DiseaseImmune Modulation Therapy in a CRIM-Positive and IgG Antibody-Positive Infant with Pompe Disease Treated with Alglucosidase Alfa: A Case ReportEffect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study.
P2860
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P2860
Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease.
description
2006 nî lūn-bûn
@nan
2006 թուականի Յուլիսին հրատարակուած գիտական յօդուած
@hyw
2006 թվականի հուլիսին հրատարակված գիտական հոդված
@hy
2006年の論文
@ja
2006年論文
@yue
2006年論文
@zh-hant
2006年論文
@zh-hk
2006年論文
@zh-mo
2006年論文
@zh-tw
2006年论文
@wuu
name
Chinese hamster ovary cell-der ...... infantile-onset Pompe disease.
@ast
Chinese hamster ovary cell-der ...... infantile-onset Pompe disease.
@en
type
label
Chinese hamster ovary cell-der ...... infantile-onset Pompe disease.
@ast
Chinese hamster ovary cell-der ...... infantile-onset Pompe disease.
@en
prefLabel
Chinese hamster ovary cell-der ...... infantile-onset Pompe disease.
@ast
Chinese hamster ovary cell-der ...... infantile-onset Pompe disease.
@en
P2093
P2860
P1476
Chinese hamster ovary cell-der ...... infantile-onset Pompe disease.
@en
P2093
Andreas Amalfitano
Anne Chun-Hui Tsai
Beth L Thurberg
Deyanira Corzo
Gail E Herman
John Waterson
Marc Nicolino
Mark Davison
Priya Sunil Kishnani
R Curtis Rogers
P2860
P356
10.1016/J.JPEDS.2006.02.035
P407
P577
2006-07-01T00:00:00Z