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

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

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

http://www.wikidata.org/entity/Q35087282

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Enzyme replacement therapy for infantile-onset Pompe disease Less Is More: Substrate Reduction Therapy for Lysosomal Storage Disorders Durable and sustained immune tolerance to ERT in Pompe disease with entrenched immune responses Hearing loss in Pompe disease revisited: results from a study of 24 children.The emerging phenotype of long-term survivors with infantile Pompe disease.The under reporting of recruitment strategies in research with children with life-threatening illnesses: A systematic review Precision medicine in the age of big data: The present and future role of large-scale unbiased sequencing in drug discovery and development.Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with 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 Cardiovascular abnormalities in late-onset Pompe disease and response to enzyme replacement therapy 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.Hypoglossal neuropathology and respiratory activity in pompe mice 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.Outcome of patients with classical infantile pompe disease receiving enzyme replacement therapy in Germany.Ocular and histologic findings in a series of children with infantile pompe disease treated with enzyme replacement therapy.Genetic counseling in Pompe disease.Immune Tolerance Strategies in Siblings with Infantile Pompe Disease-Advantages for a Preemptive Approach to High-Sustained Antibody Titers.Baseline Urinary Glucose Tetrasaccharide Concentrations in Patients with Infantile- and Late-Onset Pompe Disease Identified by Newborn Screening Defects in calcium homeostasis and mitochondria can be reversed in Pompe disease.Facial-muscle weakness, speech disorders and dysphagia are common in patients with classic infantile Pompe disease treated with enzyme therapy Public support for neonatal screening for Pompe disease, a broad-phenotype condition.CRIM-negative infantile Pompe disease: characterization of immune responses in patients treated with ERT monotherapy Familial Pompe Disease A cross-sectional single-centre study on the spectrum of Pompe disease, German patients: molecular analysis of the GAA gene, manifestation and genotype-phenotype correlations.The Changing Face of Infantile Pompe Disease: A Report of Five Patients from the UAE Hypertransaminasemia and fatal lung disease: a case report.Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational study 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 Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy.An individually, modified approach to desensitize infants and young children with Pompe disease, and significant reactions to alglucosidase alfa infusions 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 Divergent clinical outcomes of alpha-glucosidase enzyme replacement therapy in two siblings with infantile-onset Pompe disease treated in the symptomatic or pre-symptomatic state.Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease.The value of muscle biopsies in Pompe disease: identifying lipofuscin inclusions in juvenile- and adult-onset patients Clinical neurogenetics: neuropathic lysosomal storage disorders.

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P2860

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

http://www.wikidata.org/entity/Q35087282

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2009 nî lūn-bûn

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2009 թուականի Սեպտեմբերին հրատարակուած գիտական յօդուած

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2009 թվականի սեպտեմբերին հրատարակված գիտական հոդված

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2009年の論文

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2009年学术文章

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2009年学术文章

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2009年学术文章

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2009年学术文章

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2009年学术文章

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2009年學術文章

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name

Early treatment with alglucosi ...... of infants with Pompe disease

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Early treatment with alglucosi ...... of infants with Pompe disease

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Early treatment with alglucosi ...... of infants with Pompe disease

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Pediatric Research

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Early treatment with alglucosi ...... of infants with Pompe disease

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Ans Van der Ploeg

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Brian W H Tsai

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Chen-En Tsai

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Claire Morgan

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Daniel Gruskin

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Deya Corzo

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Elisa C Tsao

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Hanna Mandel

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John P Clancy

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P2860

Nonparametric Estimation from Incomplete Observations

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.

Towards a molecular therapy for glycogen storage disease type II (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.

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

Glycogen storage disease types I and II: treatment updates.

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