A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome) - Wikidata - awgldk - TriplyDB

A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)

A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)

http://www.wikidata.org/entity/Q28258072

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Enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II (Hunter syndrome)Enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II (Hunter syndrome)Enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II (Hunter syndrome)Gene therapy for mucopolysaccharidosis Imiglucerase in the treatment of Gaucher disease: a history and perspective Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy 5p deletions: Current knowledge and future directions.Therapies for the bone in mucopolysaccharidoses Drug treatment of inborn errors of metabolism: a systematic review Morquio A syndrome: diagnosis and current and future therapies CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)CNS penetration of intrathecal-lumbar idursulfase in the monkey, dog and mouse: implications for neurological outcomes of lysosomal storage disorder Respiratory and sleep disorders in mucopolysaccharidosis Clinical overview and treatment options for non-skeletal manifestations of mucopolysaccharidosis type IVA The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study.High-speed, ultrahigh resolution optical coherence tomography of the retina in Hunter syndrome.Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry.Six-Minute Walk Test demonstrates motor fatigue in spinal muscular atrophy A multicenter, open-label study evaluating safety and clinical outcomes in children (1.4-7.5 years) with Hunter syndrome receiving idursulfase enzyme replacement therapy.Growth charts for patients with Hunter syndrome.Levels of glycosaminoglycans in the cerebrospinal fluid of healthy young adults, surrogate-normal children, and Hunter syndrome patients with and without cognitive impairment Insights into Hunter syndrome from the structure of iduronate-2-sulfatase Guidelines for diagnosis and treatment of Hunter Syndrome for clinicians in Latin America.Growth patterns and the use of growth hormone in the mucopolysaccharidoses.Impact of Enzyme Replacement Therapy and Hematopoietic Stem Cell Therapy on Growth in Patients with Hunter Syndrome.Enhancement of drug delivery: enzyme-replacement therapy for murine Morquio A syndrome.Hearing loss in patients with mucopolysaccharidosis II: data from HOS - the Hunter Outcome Survey.Long-term experience with enzyme replacement therapy (ERT) in MPS II patients with a severe phenotype: an international case series.Clinical efficacy of enzyme replacement therapy in paediatric Hunter patients, an independent study of 3.5 years.The Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Questionnaire: item reduction and further validation.Correction of CNS defects in the MPSII mouse model via systemic enzyme replacement therapy.Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB.Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study.An overview of Korean patients with mucopolysaccharidosis and collaboration through the Asia Pacific MPS Network.Therapies of mucopolysaccharidosis IVA (Morquio A syndrome).Ataluren treatment of patients with nonsense mutation dystrophinopathy.Mucopolysaccharidosis I, II, and VI: Brief review and guidelines for treatment.Genistein-mediated inhibition of glycosaminoglycan synthesis, which corrects storage in cells of patients suffering from mucopolysaccharidoses, acts by influencing an epidermal growth factor-dependent pathway.Enzyme replacement therapy in newborn mucopolysaccharidosis IVA mice: early treatment rescues bone lesions?Changes in glycogen and glycosaminoglycan levels in hepatocytes of iduronate-2-sulfatase knockout mice before and after recombinant iduronate-2-sulfatase supplementation

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A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)

http://www.wikidata.org/entity/Q28258072

description

2006 nî lūn-bûn

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2006 թուականի Օգոստոսին հրատարակուած գիտական յօդուած

@hyw

2006 թվականի օգոստոսին հրատարակված գիտական հոդված

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2006年の論文

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2006年論文

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2006年論文

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2006年論文

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2006年論文

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name

A phase II/III clinical study ...... haridosis II (Hunter syndrome)

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A phase II/III clinical study ...... haridosis II (Hunter syndrome)

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A phase II/III clinical study ...... haridosis II (Hunter syndrome)

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A phase II/III clinical study ...... haridosis II (Hunter syndrome)

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A phase II/III clinical study ...... haridosis II (Hunter syndrome)

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A phase II/III clinical study ...... haridosis II (Hunter syndrome)

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A phase II/III clinical study ...... haridosis II (Hunter syndrome)

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Ana Cristina Puga

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Ann Marie Conway

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James E Wraith

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Spirometric reference values from a sample of the general U.S. population

Long-term safety and efficacy of enzyme replacement therapy for Fabry disease

Frequent deletions at Xq28 indicate genetic heterogeneity in Hunter syndrome

Incidence of Hunter's syndrome

Standardization of Spirometry, 1994 Update. American Thoracic Society

Lung volumes and forced ventilatory flows. Report Working Party Standardization of Lung Function Tests, European Community for Steel and Coal. Official Statement of the European Respiratory Society

The frequency of lysosomal storage diseases in The Netherlands.

Enzyme replacement therapy in Fabry disease: a randomized controlled trial.

ATS statement: guidelines for the six-minute walk test.

Molecular and phenotypic variation in patients with severe Hunter syndrome.

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