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Journal Article
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 Therapy, ISSN 1525-0016, 05/2018, Volume 26, Issue 5, pp. 1366 - 1374
Mucopolysaccharidosis II (MPS II) is an X-linked recessive lysosomal storage disease caused by mutations in the iduronate-2-sulfatase ( ) gene. Since IDS... 
blood-brain barrier | glycosaminoglycans | enzyme replacement therapy | iduronate-2-sulfatase | mucopolysaccharidosis II | lysosomal storage disease | transferrin receptor | HUMAN INSULIN-RECEPTOR | MEDICINE, RESEARCH & EXPERIMENTAL | 6-PHOSPHATE RECEPTOR | PRIMATE | RHESUS-MONKEYS | MONOCLONAL-ANTIBODY | REDUCES GLYCOSOAMINOGLYCANS | HUNTER-SYNDROME | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | DISEASE | GENETICS & HEREDITY | IDURONIDASE | ENZYME REPLACEMENT THERAPY | Cell Line | Tissue Distribution - drug effects | Antibodies, Monoclonal - pharmacology | Humans | Antibodies, Monoclonal - pharmacokinetics | Blood-Brain Barrier - drug effects | Receptor, IGF Type 2 - metabolism | Recombinant Fusion Proteins | Blood-Brain Barrier - metabolism | Mice, Knockout | Brain - drug effects | Brain - metabolism | Receptor, IGF Type 2 - genetics | Animals | Mucopolysaccharidosis II - metabolism | Antibodies, Monoclonal - administration & dosage | Mucopolysaccharidosis II - drug therapy | Fibroblasts - drug effects | Mucopolysaccharidosis II - genetics | Mice | Receptors, Transferrin - antagonists & inhibitors | Disease Models, Animal | Fibroblasts - metabolism | Heparan sulfate | Brain | Enzymes | Transferrin | Animal models | Intravenous administration | Glycosaminoglycans | Statistical analysis | Lysosomal storage diseases | Mucopolysaccharidosis | Monkeys & apes | Experiments | Neurological diseases | Proteins | Blood-brain barrier | Fibroblasts | Fusion protein | Drug dosages | Binding sites | Original
Journal Article
PLoS ONE, ISSN 1932-6203, 05/2016, Volume 11, Issue 5, p. e0156452
Lysosomal Storage Disorders (LSDs) are a group of metabolic syndromes, each one due to the deficit of one lysosomal enzyme. Many LSDs affect most of the organ... 
HUNTER-SYNDROME | TRANSPORT | THERAPY | DISEASES | DRUG-DELIVERY | INFLAMMATION | CONVECTION | MULTIDISCIPLINARY SCIENCES | BARRIER | CENTRAL-NERVOUS-SYSTEM | MOUSE MODELS | Albumins - chemistry | Fluorescein-5-isothiocyanate - pharmacokinetics | Nanoparticles - chemistry | Polylactic Acid-Polyglycolic Acid Copolymer | Polyglycolic Acid - pharmacology | Drug Carriers - chemistry | Lactic Acid - pharmacokinetics | Mucopolysaccharidosis II - pathology | Fluorescein-5-isothiocyanate - chemistry | Mucopolysaccharidosis II - metabolism | Nanoparticles - therapeutic use | Mucopolysaccharidosis II - genetics | Lactic Acid - pharmacology | Disease Models, Animal | Mucopolysaccharidosis I - metabolism | Fluorescein-5-isothiocyanate - pharmacology | Lactic Acid - chemistry | Mucopolysaccharidosis I - pathology | Drug Carriers - pharmacology | Blood-Brain Barrier - metabolism | Mice, Knockout | Enzyme Replacement Therapy - methods | Albumins - pharmacokinetics | Albumins - pharmacology | Animals | Polyglycolic Acid - pharmacokinetics | Mucopolysaccharidosis II - drug therapy | Mucopolysaccharidosis I - drug therapy | Polyglycolic Acid - chemistry | Mucopolysaccharidosis I - genetics | Drug Carriers - pharmacokinetics | Mice | Metabolism, Inborn errors of | Nanoparticles | Enzymes | Blood-brain barrier | Analysis | Albumin | Research | Health aspects | Therapy | Pediatrics | Animal models | Drug delivery systems | Disease | Biodegradability | Central nervous system | Disorders | Lysosomal storage diseases | Nervous system | Mucopolysaccharidosis | Neurosurgery | Molecular weight | Polylactide-co-glycolide | Biomedical materials | Low molecular weights | Rodents | Biocompatibility | Life sciences | Biodegradation | Phenotypes | Brain research | Storage | Acids | Womens health | Neurological complications | Metabolic disorders | Apoptosis
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
Journal Article
Acta Paediatrica, ISSN 0803-5253, 01/2012, Volume 101, Issue 1, pp. e42 - e47
Aim:  We present a 3‐year follow‐up of a boy with mucopolysaccharidosis type II (MPS II) who had idursulfase therapy initiated at the age of 3 months and... 
Hunter syndrome | Mucopolysaccharidosis II | Enzyme replacement therapy | Idursulfase | DIAGNOSIS | HUNTER-SYNDROME | PEDIATRICS | Enzyme Replacement Therapy | Follow-Up Studies | Mucopolysaccharidosis II - drug therapy | Humans | Child, Preschool | Diseases in Twins - drug therapy | Infant | Male | Treatment Outcome | Iduronate Sulfatase - therapeutic use | Twins | Enzymes | Medical treatment
Journal Article