Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial.
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Clinical features and predictors for disease natural progression in adults with Pompe disease: a nationwide prospective observational studyTherapeutic potential of intracerebroventricular replacement of modified human β-hexosaminidase B for GM2 gangliosidosisDisrupted autophagy undermines skeletal muscle adaptation and integrityStarch Binding Domain-containing Protein 1 Plays a Dominant Role in Glycogen Transport to Lysosomes in LiverComparative analysis of acid sphingomyelinase distribution in the CNS of rats and mice following intracerebroventricular deliveryHearing 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.Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease.The emerging phenotype of long-term survivors with infantile Pompe disease.Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease.Immunomodulatory gene therapy in lysosomal storage disordersGlycogen storage diseases: new perspectives.Autophagy in skeletal muscle: implications for Pompe diseaseHydrostatic isolated limb perfusion with adeno-associated virus vectors enhances correction of skeletal muscle in Pompe disease.Differences in the predominance of lysosomal and autophagic pathologies between infants and adults with Pompe disease: implications for therapy.Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe DiseasePompe 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.Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockadeEnhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscleEarly treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe diseaseEnzyme replacement therapy: conception, chaos and culmination.Outcome of patients with classical infantile pompe disease receiving enzyme replacement therapy in Germany.Successful induction of immune tolerance to enzyme replacement therapy in canine mucopolysaccharidosis IPreclinical Development of New Therapy for Glycogen Storage Diseases.Predicting cross-reactive immunological material (CRIM) status in Pompe disease using GAA mutations: lessons learned from 10 years of clinical laboratory testing experience.Biochemical characterization of fluorescent-labeled recombinant human alpha-L-iduronidase in vitro.Diagnosis 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 therapyImmunodominant liver-specific expression suppresses transgene-directed immune responses in murine pompe diseaseNeutralizing antibodies to therapeutic enzymes: considerations for testing, prevention and treatmentImmune 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.Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance.Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease.Glycosylation-independent lysosomal targeting of acid α-glucosidase enhances muscle glycogen clearance in pompe mice.Arrhythmias in patients receiving enzyme replacement therapy for infantile Pompe disease.Glycogen storage disease types I and II: treatment updates.
P2860
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P2860
Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial.
description
2001 nî lūn-bûn
@nan
2001 թուականի Մարտին հրատարակուած գիտական յօդուած
@hyw
2001 թվականի մարտին հրատարակված գիտական հոդված
@hy
2001年の論文
@ja
2001年論文
@yue
2001年論文
@zh-hant
2001年論文
@zh-hk
2001年論文
@zh-mo
2001年論文
@zh-tw
2001年论文
@wuu
name
Recombinant human acid alpha-g ...... f a phase I/II clinical trial.
@ast
Recombinant human acid alpha-g ...... f a phase I/II clinical trial.
@en
type
label
Recombinant human acid alpha-g ...... f a phase I/II clinical trial.
@ast
Recombinant human acid alpha-g ...... f a phase I/II clinical trial.
@en
prefLabel
Recombinant human acid alpha-g ...... f a phase I/II clinical trial.
@ast
Recombinant human acid alpha-g ...... f a phase I/II clinical trial.
@en
P2093
P1433
P1476
Recombinant human acid alpha-g ...... f a phase I/II clinical trial.
@en
P2093
Amalfitano A
Hoganson GE
Kishnani P
P304
P356
10.1097/00125817-200103000-00008
P407
P577
2001-03-01T00:00:00Z
P6179
1060304566