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Mucopolysaccharidosis

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Search Period All yearsLast 12 monthsLast 5 years Topic All topicsGeneralNutrition and dietAlternative treatment methods Type of research Any type of researchHighest Level of Evidence Only (35)Treatment guideline (2)Meta-analysis (2)Phase 3 study (16)Systematic review (9)Randomized trial (6)Clinical trial (325)Review Article (168)Early-stage study (phase 1 or 2) (13)Case report (189)Laboratory or animal study (49)Commentary or editorial (21) Studies currently recruiting participants only (33)

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Publications and studies (1372)

• Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals. (2018/01/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Multimodal image analysis of the retina in Hunter syndrome (mucopolysaccharidosis type II): Case report. (2018/01/01) ♡
• High-Throughput Screen Fails to Identify Compounds That Enhance Residual Enzyme Activity of Mutant N-Acetyl-α-Glucosaminidase in Mucopolysaccharidosis Type IIIB. (2018/01/01) ♡
• Optimization of alginate microcapsules containing cells overexpressing α-l-iduronidase using Box-Behnken design. (2018/01/01) ♡
• The clinical and genetic Spectrum of Maroteaux-Lamy syndrome (Mucopolysaccharidosis VI) in the Eastern Province of Saudi Arabia. (2018/01/01) ♡
• Gene editing of MPS I human fibroblasts by co-delivery of a CRISPR/Cas9 plasmid and a donor oligonucleotide using nanoemulsions as nonviral carriers. (2018/01/01) ♡
• Cardiopulmonary Exercise Testing Reflects Improved Exercise Capacity in Response to Treatment in Morquio A Patients: Results of a 52-Week Pilot Study of Two Different Doses of Elosulfase Alpha. (2018/01/01) ♡
• Cyclodextrin encapsulation of daidzein and genistein by grinding: implication on the glycosaminoglycan accumulation in mucopolysaccharidosis type II and III fibroblasts. (2018/01/01) ♡
• Mucopolysaccharidosis at play? (2018/01/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Neurological manifestations of lysosomal disorders and emerging therapies targeting the CNS. (2018/01/01) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Enzyme replacement therapies: what is the best option? (2018/01/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Arthropathy-like findings and a carpal tunnel syndrome as the presenting features of Scheie syndrome: Three cases from the same family. (2018/01/01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Effects of Growth Hormone in Chronically Ill Children (2018-12-14) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Intrathecal Enzyme Replacement for Hurler Syndrome (2018-12-03) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD) (2018-11-28) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome (2018-09-27) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181 (2018-09-25) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety and Dose Ranging Study of Insulin Receptor MOAB-IDS Fusion Protein in Patients With Hunter Syndrome (2018-09-18) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB (2018-09-13) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Clinical Trial of Growth Hormone in MPS I, II, and VI (2018-09-05) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB) (2018-08-21) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Open Label Study in Previously Studied, SBC-103 Treatment Naive MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously (2018-04-17) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study) (2018-04-11) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Allogenetic Bone Marrow Transplant for Inherited Metabolic Disorders (2018-02-05) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety and Effect of L. Reuters on Biomarkers of Inflammation in Healthy Infants With Colic (2018-01-30) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Ultrasound Findings of Finger, Wrist, and Knee Joints in Mucopolysaccharidosis (2018-01-24) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative Diagnosis (2018-01-03) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Natural history of Morquio A patient with tracheal obstruction from birth to death. (2017/12/22) ♡
• Quantification of the enzyme activities of iduronate-2-sulfatase, N-acetylgalactosamine-6-sulfatase and N-acetylgalactosamine-4-sulfatase using liquid chromatography-tandem mass spectrometry. (2017/12/21) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Widespread Vasculopathy in a Patient with Morquio A Syndrome. (2017/12/19) ♡
• Guanidinylated Neomycin Conjugation Enhances Intranasal Enzyme Replacement in the Brain. (2017/12/06) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Preclinical and clinical advances in transposon-based gene therapy. (2017/12/05) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Carpal tunnel syndrome in mucopolysaccharidosis I: a registry-based cohort study. (2017/12/01) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Carpal tunnel syndrome in mucopolysaccharidosis: the value of rare disease registries. (2017/12/01) ♡
• Beta-glucuronidase activity in dried blood spots: Reduced technique with biochemical parameters determined. (2017/12/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Outcome of Combined Mitral and Aortic Valve Replacement in Adults With Mucopolysaccharidosis (the Hurler Syndrome). (2017/12/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Pathophysiology, evaluation, and management of sleep disorders in the mucopolysaccharidoses. (2017/12/01) ♡
• Urinary metabolic phenotyping of mucopolysaccharidosis type I combining untargeted and targeted strategies with data modeling. (2017/12/01) ♡
• Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy. (2017/12/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Phase I and II clinical trials for the mucopolysaccharidoses. (2017/12/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Developmental and behavioral aspects of mucopolysaccharidoses with brain manifestations - Neurological signs and symptoms. (2017/12/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Quantitative neuroimaging in mucopolysaccharidoses clinical trials. (2017/12/01) ♡
• N-glycan structures and downstream mannose phosphorylation of plant recombinant human alpha-L-iduronidase: toward development of enzyme replacement therapy for mucopolysaccharidosis I. (2017/12/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Assessments of neurocognitive and behavioral function in the mucopolysaccharidoses (2017/12/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Treatment of brain disease in mucopolysaccharidoses. (2017/12/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Surgical management of neurological manifestations of mucopolysaccharidosis disorders. (2017/12/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Practical management of behavioral problems in mucopolysaccharidoses disorders. (2017/12/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Epilepsy in mucopolysaccharidosis disorders. (2017/12/01) ♡
• Whole Body and CNS Biodistribution of RhHNS in Cynomolgus Monkeys after Intrathecal Lumbar Administration: Treatment Implications for Patients with MPS IIIA. (2017/12/01) ♡
• Deleting the mouse Hsd17b1 gene results in a hypomorphic Naglu allele and a phenotype mimicking a lysosomal storage disease. (2017/11/27) ♡

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