The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease. - Wikidata - wikimedia - TriplyDB

The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease.

The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease.

http://www.wikidata.org/entity/Q34629805

about

Enzyme replacement therapy for infantile-onset Pompe disease Durable and sustained immune tolerance to ERT in Pompe disease with entrenched immune responses The emerging phenotype of long-term survivors with infantile Pompe disease.Pompe disease: from pathophysiology to therapy and back again 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.Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade Enzyme therapy and immune response in relation to CRIM status: the Dutch experience in classic infantile Pompe disease.Proteasome Inhibitor Bortezomib Enhances the Activity of Multiple Mutant Forms of Lysosomal α-Glucosidase in Pompe Disease Outcome of patients with classical infantile pompe disease receiving enzyme replacement therapy in Germany.Immunogenicity of idursulfase and clinical outcomes in very young patients (16 months to 7.5 years) with mucopolysaccharidosis II (Hunter syndrome).A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I.Preclinical Development of New Therapy for Glycogen Storage Diseases.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.Immunogenicity to therapeutic proteins: impact on PK/PD and efficacy.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.Clinical Laboratory Experience of Blood CRIM Testing in Infantile Pompe Disease Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease.Mucopolysaccharidosis type VI phenotypes-genotypes and antibody response to galsulfase.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 study Transcription factor EB (TFEB) is a new therapeutic target for Pompe disease WITHDRAWN: Clearance of lysosomal glycogen accumulation by Transcription factor EB (TFEB) in muscle cells from lysosomal alpha-glucosidase deficient mice.Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy.Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease.Atypical immunologic response in a patient with CRIM-negative Pompe disease.Persistence of high sustained antibodies to enzyme replacement therapy despite extensive immunomodulatory therapy in an infant with Pompe disease: need for agents to target antibody-secreting plasma cells.Bortezomib in the rapid reduction of high sustained antibody titers in disorders treated with therapeutic protein: lessons learned from Pompe disease Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease.Successful Desensitisation in a Patient with CRIM-Positive Infantile-Onset Pompe Disease.Immune tolerance induction for laronidase treatment in mucopolysaccharidosis I.Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction.Enzyme replacement therapy for Pompe disease.Infantile Pompe disease on ERT: update on clinical presentation, musculoskeletal management, and exercise considerations.Treatment of lysosomal storage disorders: successes and challenges.Strategic characterization of anti-drug antibody responses for the assessment of clinical relevance and impact.Dendritic cells and pluripotency: unlikely allies in the pursuit of immunotherapy.Response of 33 UK patients with infantile-onset Pompe disease to enzyme replacement therapy.

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P2860

The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease.

http://www.wikidata.org/entity/Q34629805

description

2011 nî lūn-bûn

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2011 թուականի Օգոստոսին հրատարակուած գիտական յօդուած

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2011 թվականի օգոստոսին հրատարակված գիտական հոդված

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2011年の論文

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P2860

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

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.

Human immune response to recombinant human proteins.

A randomized study of alglucosidase alfa in late-onset Pompe's disease.

Pre- and postnatal pathology, enzyme treatment, and unresolved issues in five lysosomal disorders.

Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease

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

Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance.

Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I.

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