Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease. - Wikidata - awgldk - TriplyDB

Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease.

Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease.

http://www.wikidata.org/entity/Q51768443

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New insights into therapeutic options for Pompe disease The 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 disease Durable and sustained immune tolerance to ERT in Pompe disease with entrenched immune responses Rasch analysis of the Pediatric Evaluation of Disability Inventory-computer adaptive test (PEDI-CAT) item bank for children and young adults with spinal muscular atrophy.Albuterol as an adjunctive treatment to enzyme replacement therapy in infantile-onset Pompe disease.Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease.Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease Fiber type conversion by PGC-1α activates lysosomal and autophagosomal biogenesis in both unaffected and Pompe skeletal muscle.Oral delivery of bioencapsulated coagulation factor IX prevents inhibitor formation and fatal anaphylaxis in hemophilia B mice.Pompe disease: from pathophysiology to therapy and back again 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.Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease Suppression of autophagy permits successful enzyme replacement therapy in a lysosomal storage disorder--murine Pompe disease.The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease.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.Myostatin and insulin-like growth factor I: potential therapeutic biomarkers for pompe disease Pompe disease gene therapy.Hypoglossal neuropathology and respiratory activity in pompe mice Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease Enzyme therapy and immune response in relation to CRIM status: the Dutch experience in classic infantile Pompe disease.Outcome of patients with classical infantile pompe disease receiving enzyme replacement therapy in Germany.Preclinical Development of New Therapy for Glycogen Storage Diseases.β2 Agonists enhance the efficacy of simultaneous enzyme replacement therapy in murine Pompe disease.BAFF blockade prevents anti-drug antibody formation in a mouse model of Pompe disease Predicting cross-reactive immunological material (CRIM) status in Pompe disease using GAA mutations: lessons learned from 10 years of clinical laboratory testing experience.Diagnosis and treatment of late-onset Pompe disease in the Middle East and North Africa region: consensus recommendations from an expert group Immune 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 therapy CRIM-negative infantile Pompe disease: characterization of immune responses in patients treated with ERT monotherapy Synergistic Efficacy from Gene Therapy with Coreceptor Blockade and a β2-Agonist in Murine Pompe Disease.Immune Modulation Therapy in a CRIM-Positive and IgG Antibody-Positive Infant with Pompe Disease Treated with Alglucosidase Alfa: A Case Report Adjunctive β2-agonists reverse neuromuscular involvement in murine Pompe disease.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.Hypertransaminasemia and fatal lung disease: a case report.Salmeterol enhances the cardiac response to gene therapy in Pompe disease.Diaphragm Pacing as a Rehabilitative Tool for Patients With Pompe Disease Who Are Ventilator-Dependent: Case Series.Phase 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.

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Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease.

http://www.wikidata.org/entity/Q51768443

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Clinical outcomes after long-t ...... n with advanced Pompe disease.

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Clinical outcomes after long-t ...... n with advanced Pompe disease.

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Clinical outcomes after long-t ...... n with advanced Pompe disease.

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Clinical outcomes after long-t ...... n with advanced Pompe disease.

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Clinical outcomes after long-t ...... n with advanced Pompe disease.

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Clinical outcomes after long-t ...... n with advanced Pompe disease.

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Clinical outcomes after long-t ...... n with advanced Pompe disease.

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Barry Byrne

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Brigitte Chabrol

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Claire Morgan

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Georgianne L Arnold

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P2860

Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease

Hearing loss in infantile Pompe's disease and determination of underlying pathology in the knockout mouse

Glycogen stored in skeletal but not in cardiac muscle in acid alpha-glucosidase mutant (Pompe) mice is highly resistant to transgene-encoded human enzyme

Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease.

Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial.

Enzyme therapy for pompe disease with recombinant human alpha-glucosidase from rabbit milk.

The natural course of infantile Pompe's disease: 20 original cases compared with 133 cases from the literature.

Pompe disease in infants and children.

Left ventricular myocardial mass determined by cross-sectional echocardiography in normal newborns, infants, and children.

Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk.

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