Recombinant human alpha-glucosidase from rabbit milk in Pompe patients.
about
Clinical features and predictors for disease natural progression in adults with Pompe disease: a nationwide prospective observational studyN-glycans of recombinant human acid alpha-glucosidase expressed in the milk of transgenic rabbits.Albuterol as an adjunctive treatment to enzyme replacement therapy in infantile-onset Pompe disease.Recombinant human plasma phospholipid transfer protein (PLTP) to prevent bacterial growth and to treat sepsis.Human Alpha Galactosidases Transiently Produced in Nicotiana benthamiana Leaves: New Insights in Substrate Specificities with Relevance for Fabry DiseaseAutophagy in skeletal muscle: implications for Pompe diseaseThe pharmacological chaperone AT2220 increases recombinant human acid α-glucosidase uptake and glycogen reduction in a mouse model of Pompe disease.Pompe disease diagnosis and management guideline.The mousetrap: what we can learn when the mouse model does not mimic the human disease.Cardiovascular abnormalities in late-onset Pompe disease and response to enzyme replacement therapyEnzyme therapy for Pompe disease: from science to industrial enterprise.Survival 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.Burden of illness of Pompe disease in patients only receiving supportive care.Outcome of patients with classical infantile pompe disease receiving enzyme replacement therapy in Germany.A chaperone enhances blood α-glucosidase activity in Pompe disease patients treated with enzyme replacement therapy.The molecular era of myology.Electrocardiographic and other cardiac anomalies in beta-glucuronidase-null mice corrected by nonablative neonatal marrow transplantationPredicting cross-reactive immunological material (CRIM) status in Pompe disease using GAA mutations: lessons learned from 10 years of clinical laboratory testing experience.Postmortem Findings and Clinical Correlates in Individuals with Infantile-Onset Pompe DiseaseFacial-muscle weakness, speech disorders and dysphagia are common in patients with classic infantile Pompe disease treated with enzyme therapyA conceptual disease model for adult Pompe disease.Lack of robust satellite cell activation and muscle regeneration during the progression of Pompe disease.Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study.Exercise testing in late-onset glycogen storage disease type II patients undergoing enzyme replacement therapy.iPS cell modeling of cardiometabolic diseasesDelivery of lysosomal enzymes for therapeutic use: glucocerebrosidase as an example.Glycogen storage disease types I and II: treatment updates.Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational studyNovel therapeutic targets for the treatment of Fabry disease.Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center studySlow, progressive myopathy in neonatally treated patients with infantile-onset Pompe disease: a muscle magnetic resonance imaging study.Progress and problems when considering gene therapy for GSD-II.Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants.Cardiac remodeling after enzyme replacement therapy with acid alpha-glucosidase for infants with Pompe disease.A review of treatment of Pompe disease in infantsTherapeutic approaches in glycogen storage disease type II/Pompe Disease.Temporal neuropathologic and behavioral phenotype of 6neo/6neo Pompe disease mice.Biochemical and pharmacological characterization of different recombinant acid alpha-glucosidase preparations evaluated for the treatment of Pompe disease.Pharmacological small molecules for the treatment of lysosomal storage disorders.
P2860
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P2860
Recombinant human alpha-glucosidase from rabbit milk in Pompe patients.
description
2000 nî lūn-bûn
@nan
2000年の論文
@ja
2000年学术文章
@wuu
2000年学术文章
@zh
2000年学术文章
@zh-cn
2000年学术文章
@zh-hans
2000年学术文章
@zh-my
2000年学术文章
@zh-sg
2000年學術文章
@yue
2000年學術文章
@zh-hant
name
Recombinant human alpha-glucosidase from rabbit milk in Pompe patients.
@en
Recombinant human alpha-glucosidase from rabbit milk in Pompe patients.
@nl
type
label
Recombinant human alpha-glucosidase from rabbit milk in Pompe patients.
@en
Recombinant human alpha-glucosidase from rabbit milk in Pompe patients.
@nl
prefLabel
Recombinant human alpha-glucosidase from rabbit milk in Pompe patients.
@en
Recombinant human alpha-glucosidase from rabbit milk in Pompe patients.
@nl
P2093
P1433
P1476
Recombinant human alpha-glucosidase from rabbit milk in Pompe patients.
@en
P2093
A Cromme-Dijkhuis
A J Reuser
A T Van der Ploeg
H Van den Hout
M C Loonen
P304
P356
10.1016/S0140-6736(00)02533-2
P407
P577
2000-07-01T00:00:00Z