A study of intrathecal enzyme replacement for cognitive decline in mucopolysaccharidosis I (original) (raw)
Safety Evaluation of Chronic Intrathecal Administration of Idursulfase-IT in Cynomolgus Monkeys
michael heartlein
Toxicologic Pathology, 2011
View PDFchevron_right
Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study
Matilde ruiz garcia
View PDFchevron_right
Journal Pre-proof Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II
Matilde ruiz garcia
View PDFchevron_right
A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II
Christian Hendriksz
Genetics in medicine : official journal of the American College of Medical Genetics, 2015
View PDFchevron_right
CNS Penetration of Intrathecal-Lumbar Idursulfase in the Monkey, Dog and Mouse: Implications for Neurological Outcomes of Lysosomal Storage Disorder
michael heartlein
PLoS ONE, 2012
View PDFchevron_right
Biodistribution and pharmacodynamics of recombinant human alpha-l-iduronidase (rhIDU) in mucopolysaccharidosis type I-affected cats following multiple intrathecal administrations
Raquel Walton
Molecular Genetics and Metabolism, 2011
View PDFchevron_right
Effect of systemic high dose enzyme replacement therapy on the improvement of CNS defects in a mouse model of mucopolysaccharidosis type II
Ah-ra Ko
Orphanet journal of rare diseases, 2015
View PDFchevron_right
Uptake of recombinant iduronate-2-sulfatase into neuronal and glial cells in vitro
Aurora Daniele
Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease, 2002
View PDFchevron_right
Intrathecal enzyme replacement therapy: Successful treatment of brain disease via the cerebrospinal fluid
Steven Le
Molecular Genetics and Metabolism, 2007
View PDFchevron_right
Effect of neonatal administration of a retroviral vector expressing α-l-iduronidase upon lysosomal storage in brain and other organs in mucopolysaccharidosis I mice
Katherine Ponder
Molecular Genetics and Metabolism, 2007
View PDFchevron_right
Effect of high dose, repeated intra-cerebrospinal fluid injection of sulphamidase on neuropathology in mucopolysaccharidosis type IIIA mice
Helen Beard
Genes, Brain and Behavior, 2008
View PDFchevron_right
Intrathecal enzyme replacement therapy reverses cognitive decline in mucopolysaccharidosis type I
Alia Ahmed
American journal of medical genetics. Part A, 2017
View PDFchevron_right
Replacing the Enzyme -L-Iduronidase at Birth Ameliorates Symptoms in the Brain and Periphery of Dogs with Mucopolysaccharidosis Type I
A. Fales-Williams
Science Translational Medicine, 2010
View PDFchevron_right
Preclinical dose ranging studies for enzyme replacement therapy with idursulfase in a knock-out mouse model of MPS II
Antony Emmanuel Camacho Garcia
Molecular Genetics and Metabolism, 2007
View PDFchevron_right
Profile of idursulfase for the treatment of Hunter syndrome
Ferdinando Ceravolo
Research and Reports in Endocrine Disorders, 2015
View PDFchevron_right
Intravenous delivery of a chemically modified sulfamidase efficiently reduces heparan sulfate storage and brain pathology in mucopolysaccharidosis IIIA mice
Agneta Tjernberg
Molecular Genetics and Metabolism Reports, 2019
View PDFchevron_right
Successful desensitization to idursulfase in a patient with type II mucopolysaccharidosis (Hunter syndrome)
Carlos Serrano
Journal of investigational allergology & clinical immunology, 2011
View PDFchevron_right
High-dose enzyme replacement therapy attenuates cerebroventriculomegaly in a mouse model of mucopolysaccharidosis type II
Chihwa Kim
Journal of Human Genetics, 2013
View PDFchevron_right
Intracerebroventricular dosing of N-sulfoglucosamine sulfohydrolase in mucopolysaccharidosis IIIA mice reduces markers of brain lysosomal dysfunction
Terri Christianson
Journal of Biological Chemistry, 2022
View PDFchevron_right
Penetration, diffusion, and uptake of recombinant human α-l-iduronidase after intraventricular injection into the rat brain
Steven Jungles
Molecular Genetics and Metabolism, 2005
View PDFchevron_right
α-L-iduronidase therapy for mucopolysaccharidosis type I
Paul Orchard
Biologics: targets & therapy, 2008
View PDFchevron_right
Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice
Gordon Watson
Gene Therapy, 2006
View PDFchevron_right
A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)
Roberto Giugliani
Genetics in Medicine, 2006
View PDFchevron_right
The Effect of Recombinant Human Iduronate-2-Sulfatase (Idursulfase) on Growth in Young Patients with Mucopolysaccharidosis Type II
Anna Tylki-szymanska
PLoS ONE, 2014
View PDFchevron_right
Glycosaminoglycan storage in neuroanatomical regions of mucopolysaccharidosis I dogs following intrathecal recombinant human iduronidase
Catalina Guerra
APMIS, 2011
View PDFchevron_right
Comparative study of idursulfase beta and idursulfase in vitro and in vivo
Chihwa Kim
Journal of Human Genetics, 2016
View PDFchevron_right
Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene
Roland Gunther
Molecular therapy : the journal of the American Society of Gene Therapy, 2004
View PDFchevron_right
Correction of Metabolic, Craniofacial, and Neurologic Abnormalities in MPS I Mice Treated at Birth with Adeno-associated Virus Vector Transducing the Human α-L-Iduronidase Gene
Walter Low
Molecular Therapy, 2004
View PDFchevron_right
Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy
Sara Marcó, Sandra Motas, Luca Maggioni
Journal of Clinical Investigation, 2013
View PDFchevron_right
Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human α-L-iduronidase (laronidase)
Kenneth Berger
The Journal of Pediatrics, 2004
View PDFchevron_right
An Improved Adeno-Associated Virus Vector for Neurological Correction of the Mouse Model of Mucopolysaccharidosis IIIA
Jessica Taylor
2019
View PDFchevron_right
RTB lectin-mediated delivery of lysosomal α-l-iduronidase mitigates disease manifestations systemically including the central nervous system
Chester Whitley
Molecular genetics and metabolism, 2018
View PDFchevron_right