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Journal Article
Journal Article
PLoS ONE, ISSN 1932-6203, 01/2012, Volume 7, Issue 1, p. e30341
A major challenge for the treatment of many central nervous system (CNS) disorders is the lack of convenient and effective methods for delivering biological... 
MUCOPOLYSACCHARIDOSIS-II | TRANSFERRIN RECEPTOR | MULTIDISCIPLINARY SCIENCES | ADENOASSOCIATED VIRUS VECTOR | CENTRAL-NERVOUS-SYSTEM | MONOCLONAL-ANTIBODY | II HUNTER-SYNDROME | CEREBROSPINAL-FLUID | BLOOD-BRAIN-BARRIER | ENZYME REPLACEMENT THERAPY | GROWTH FACTOR-II/MANNOSE-6-PHOSPHATE RECEPTOR | Immunohistochemistry | Recombinant Proteins - therapeutic use | Neurons - pathology | Oligodendroglia - metabolism | Central Nervous System - metabolism | Iodine Radioisotopes | Spinal Cord - drug effects | Spinal Cord - metabolism | Species Specificity | Humans | Central Nervous System - pathology | Macaca fascicularis | Recombinant Proteins - pharmacokinetics | Positron-Emission Tomography | Tissue Distribution | Lysosomes - metabolism | Mucopolysaccharidosis II - metabolism | Oligodendroglia - drug effects | Spinal Cord - pathology | Mucopolysaccharidosis II - genetics | Iduronate Sulfatase - administration & dosage | Neurons - metabolism | Neurons - drug effects | Injections, Spinal | Recombinant Proteins - administration & dosage | Mice, Knockout | Oligodendroglia - pathology | Enzyme Replacement Therapy - methods | Animals | Mucopolysaccharidosis II - drug therapy | Dogs | Central Nervous System - drug effects | Iduronate Sulfatase - genetics | Mice | Iduronate Sulfatase - therapeutic use | Enzymes | Biological products | Gene therapy | Neurons | Brain | Nuclear medicine | Spinal cord | Drug delivery systems | Intravenous administration | Glycosaminoglycans | Central nervous system | Medical services | Parenchyma | Lysosomes | Mucopolysaccharidosis | Insulin-like growth factors | Cerebrospinal fluid | Molecular weight | Proteins | Biological effects | Rodents | Animal tissues | Oligodendrocytes | Primates | Recombinant | Medical instruments | Insulin | Biological activity | Hospitals | Algorithms | Storage | Biological warfare | Alzheimers disease
Journal Article
Molecular Genetics and Metabolism, ISSN 1096-7192, 2007, Volume 90, Issue 3, pp. 329 - 337
To evaluate the safety and explore the efficacy of idursulfase (recombinant human iduronate-2-sulfatase) treatment for mucopolysaccharidosis II (MPS II).... 
Hunter syndrome | MPS II | Iduronate-2-sulfatase | Glycosaminoglycans | Enzyme replacement therapy | Idursulfase | Lysosomal storage disorder | Mucopolysaccharidosis II | glycosaminoglycans | idursulfase | MEDICINE, RESEARCH & EXPERIMENTAL | OBSTRUCTIVE SLEEP-APNEA | enzyme replacement therapy | 6-MINUTE WALK TEST | BONE-MARROW-TRANSPLANTATION | FABRY-DISEASE | N-ACETYLGALACTOSAMINE 4-SULFATASE | lysosomal storage disorder | DIMETHYLMETHYLENE BLUE | CHILDREN | iduronate-2-sulfatase | mucopolysaccharidosis II | ENDOCRINOLOGY & METABOLISM | GENETICS & HEREDITY | TERM-FOLLOW-UP | SCREENING-PROCEDURE | Recombinant Proteins - therapeutic use | Liver - pathology | Humans | Male | Spleen - drug effects | Recombinant Proteins - adverse effects | Joints - physiopathology | Mucopolysaccharidosis II - pathology | Iduronate Sulfatase - immunology | Immunoglobulin G - biosynthesis | Liver - drug effects | Mucopolysaccharidosis II - physiopathology | Adult | Iduronate Sulfatase - administration & dosage | Safety | Spleen - pathology | Child | Glycosaminoglycans - urine | Double-Blind Method | Joints - drug effects | Recombinant Proteins - administration & dosage | Recombinant Proteins - immunology | Mucopolysaccharidosis II - drug therapy | Adolescent | Infusions, Intravenous | Respiratory Function Tests | Iduronate Sulfatase - therapeutic use | Iduronate Sulfatase - adverse effects | Clinical trials | Product development | Biopharmaceutics
Journal Article
Molecular Genetics and Metabolism, ISSN 1096-7192, 02/2014, Volume 111, Issue 2, pp. 63 - 72
The mucopolysaccharidoses (MPS), a group of rare genetic disorders caused by defects in glycosaminoglycan (GAG) catabolism, are progressive, multi-systemic... 
Pre-symptomatic | Mucopolysaccharidosis | Laronidase | Enzyme replacement therapy | Galsulfase | Idursulfase | MEDICINE, RESEARCH & EXPERIMENTAL | HURLER-SYNDROME | JOINT DISEASE | FOLLOW-UP | N-ACETYLGALACTOSAMINE 4-SULFATASE | LYSOSOMAL STORAGE DISORDERS | MPS-I | HUNTER OUTCOME SURVEY | ENDOCRINOLOGY & METABOLISM | GENETICS & HEREDITY | L-IDURONIDASE LARONIDASE | HEPARAN-SULFATE | STEM-CELL TRANSPLANTATION | N-Acetylgalactosamine-4-Sulfatase - therapeutic use | Recombinant Proteins - therapeutic use | Humans | Child, Preschool | Secondary Prevention | Dysostoses - physiopathology | Joints - physiopathology | Respiratory System - enzymology | Heart Valves - drug effects | Mucopolysaccharidoses - complications | Mucopolysaccharidoses - drug therapy | Enzyme Replacement Therapy | Heart Valves - enzymology | Dysostoses - enzymology | Joints - enzymology | Mucopolysaccharidoses - physiopathology | Glycosaminoglycans - metabolism | Joints - drug effects | Mucopolysaccharidoses - enzymology | Dysostoses - complications | Clinical Trials as Topic | Dysostoses - drug therapy | Respiratory System - drug effects | Respiratory System - physiopathology | Iduronate Sulfatase - therapeutic use | Heart Valves - physiopathology | Enzymes | Care and treatment | Glycosaminoglycans | Physiological aspects | Health aspects | Biopharmaceutics
Journal Article
Journal Article
Journal Article
Allergy, ISSN 0105-4538, 06/2013, Volume 68, Issue 6, pp. 796 - 802
Journal Article