A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II. - Wikidata - awgldk - TriplyDB

A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II.

A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II.

http://www.wikidata.org/entity/Q41122864

about

Enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II (Hunter syndrome)CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)Comparative studies of vertebrate iduronate 2-sulfatase (IDS) genes and proteins: evolution of A mammalian X-linked gene Safety Evaluation of CNS Administered Biologics-Study Design, Data Interpretation, and Translation to the Clinic.Levels of glycosaminoglycans in the cerebrospinal fluid of healthy young adults, surrogate-normal children, and Hunter syndrome patients with and without cognitive impairment Newborn Screening for Lysosomal Storage Diseases: A Concise Review of the Literature on Screening Methods, Therapeutic Possibilities and Regional Programs Gene therapy for lysosomal storage disorders: a good start.Enzyme replacement therapy and beyond-in memoriam Roscoe O. Brady, M.D. (1923-2016).Safety of laronidase delivered into the spinal canal for treatment of cervical stenosis in mucopolysaccharidosis I.Biodistribution of Idursulfase Formulated for Intrathecal Use (Idursulfase-IT) in Cynomolgus Monkeys after Intrathecal Lumbar Administration.Protease-resistant modified human β-hexosaminidase B ameliorates symptoms in GM2 gangliosidosis model The levels of urinary glycosaminoglycans of patients with attenuated and severe type of mucopolysaccharidosis II determined by liquid chromatography-tandem mass spectrometry.Development of idursulfase therapy for mucopolysaccharidosis type II (Hunter syndrome): the past, the present and the future.Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration.Treatment of mucopolysaccharidosis type II (Hunter syndrome): results from a systematic evidence review.Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications.Survival in idursulfase-treated and untreated patients with mucopolysaccharidosis type II: data from the Hunter Outcome Survey (HOS).Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID.Mucopolysaccharidosis type I, II and VI and response to enzyme replacement therapy: Results from a single-center case series study.The use of port-a-caths in adult patients with Lysosomal Storage Disorders receiving Enzyme Replacement Therapy-one centre experience.Phase I and II clinical trials for the mucopolysaccharidoses.Presentation and Treatments for Mucopolysaccharidosis Type II (MPS II; Hunter Syndrome).Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy.Lipid biomarkers for the peroxisomal and lysosomal disorders: their formation, metabolism and measurement.Whole Body and CNS Biodistribution of rhHNS in Cynomolgus Monkeys after Intrathecal Lumbar Administration: Treatment Implications for Patients with MPS IIIA.Workshop Proceedings: Streamlined Development of Safety Assessment Programs Supporting Orphan/Rare Diseases-Are We There Yet?A comparison study of bioanalytical methods for characterization of anti-idursulfase antibodies.Effectiveness of Early Hematopoietic Stem Cell Transplantation in Preventing Neurocognitive Decline in Mucopolysaccharidosis Type II: A Case Series.A Blood-Brain-Barrier-Penetrating Anti-human Transferrin Receptor Antibody Fusion Protein for Neuronopathic Mucopolysaccharidosis II.Fast, sensitive method for trisaccharide biomarker detection in mucopolysaccharidosis type 1.Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after 52 weeks of intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein (valanafusp alpha): an open label phase 1-2 t Targeting Root Cause by Systemic scAAV9-h Gene Delivery: Functional Correction and Reversal of Severe MPS II in Mice

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A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II.

http://www.wikidata.org/entity/Q41122864

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Genetics in Medicine

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A phase I/II study of intrathe ...... vere mucopolysaccharidosis II.

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Ann J Barbier

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Joseph Muenzer

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Kenneth Sciarappa

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Mary Ann Mascelli

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Nan Wang

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Suresh Vijayaraghavan

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Victor Perry

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Zheng Fan

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P2860

Human liver iduronate-2-sulphatase. Purification, characterization and catalytic properties

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

CNS penetration of intrathecal-lumbar idursulfase in the monkey, dog and mouse: implications for neurological outcomes of lysosomal storage disorder

Behaviour in mucopolysaccharide disorders.

The defect in the Hunter syndrome: deficiency of sulfoiduronate sulfatase.

Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome).

Multidisciplinary management of Hunter syndrome.

Early clinical markers of central nervous system involvement in mucopolysaccharidosis type II.

The role of enzyme replacement therapy in severe Hunter syndrome-an expert panel consensus

Physiology of the intrathecal bolus: the leptomeningeal route for macromolecule and particle delivery to CNS.

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