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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 (1371)

• Commentary or editorialiAn expert's opinion or commentary, not new research. Modified umbilical cord-blood transplantation for pediatric patients with mucopolysaccharidosis. (2023/01/01) ♡
• Activities of (Poly)phenolic Antioxidants and Other Natural Autophagy Modulators in the Treatment of Sanfilippo Disease: Remarkable Efficacy of Resveratrol in Cellular and Animal Models. (2023/01/01) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Morquio-Brailsford Syndrome: Analysis of Bone Mass and Relevant Clinical Considerations. (2023/01/01) ♡
• Dilated Optic Nerve Sheath in Mucopolysaccharidosis I: Common and Not Necessarily High Intracranial Pressure. (2023/01/01) ♡
• Synthesis and Evaluation of Novel Triaryl Derivatives with Readthrough-Inducing Activity. (2023/01/01) ♡
• Mucopolysaccharidosis II with neonatal respiratory distress syndrome and bronchopulmonary dysplasia. (2023/01/01) ♡
• Discovery of Novel Chromenopyridine Derivatives as Readthrough-Inducing Drugs. (2023/01/01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Efficacy of BIS Monitoring in Deep Hypnotic State (2023-12-20) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I (2023-10-31) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Natural History Study in Children With a Type II Collagen Disorder With Short Stature (2023-10-02) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation (2023-09-15) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701) (2023-09-15) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Core Outcome Set for Head, Neck and Respiratory Disease in Mucopolysaccharidosis II (2023-09-01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II (2023-08-28) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Assessment of Artificial Intelligence for Treatment Decision Recommendation of Adult Skeletal Class III Patients (2023-08-23) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE (2023-08-09) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease (2023-07-25) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Decoding the Genetic Landscape of Skeletal Diseases (2023-05-25) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Effect of Inhalation of Low Dose CO2 on Exercise Performance at High Altitude (2023-05-18) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Identification and Characterization of Bone-related Genetic Variants in Families (2023-04-19) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders (2023-04-12) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety and Efficacy of Encapsulated Allogeneic MPS-1 Therapy (2023-04-11) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Validation and Reliability of Iris Cameras in Mucopolysaccharidoses (2023-03-22) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety and Dose Ranging Study of Human Insulin Receptor MAb-IDUA Fusion Protein in Adults and Children With MPS I (2023-03-15) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I (2023-03-15) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I (2023-02-21) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Effectiveness Verification of Using Hydrogen Inhalation to Improve Fatigue, Pain and Quality of Life in Cancer Patients (2023-02-14) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Biomarker for Maroteaux-Lamy Disease (BioMaroteaux) (2023-02-13) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Biomarker for Gangliosidosis: BioGM1/BioGM2 (BioGM1/GM2) (2023-02-13) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Biomarker for Morquio Disease (BioMorquio) (2023-02-13) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Biomarker for Sly Disease (MPS VII) (BioSly) (2023-02-13) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Biomarker for Hurler Disease (BioHurler) (2023-02-13) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Biomarkers for Hunter Syndrome (2023-02-10) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I (2023-01-26) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Treatment Extension Study of Mucopolysaccharidosis Type IIIB (2023-01-18) ♡
• 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. Mucopolysaccharidoses: Cellular Consequences of Glycosaminoglycans Accumulation and Potential Targets. (2022/12/28) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Murine Fibroblasts and Primary Hepatocytes as Tools When Studying the Efficacy of Potential Therapies for Mucopolysaccharidosis Type I. (2022/12/28) ♡
• 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. Mucopolysaccharidosis: What Pediatric Rheumatologists and Orthopedics Need to Know. (2022/12/27) ♡
• 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. Burden of Surgical Treatment for the Management of Cervical Myelopathy in Mucopolysaccharidoses: A Systematic Review. (2022/12/26) ♡
• Effects of gentamicin inducing readthrough premature stop codons: A study of alpha-L-iduronidase nonsense variants in COS-7 Cells. (2022/12/25) ♡
• Understanding the challenges, unmet needs, and expectations of mucopolysaccharidoses I, II and VI patients and their caregivers in France: a survey study. (2022/12/23) ♡
• 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. Diagnosis and Emerging Treatment Strategies for Mucopolysaccharidosis VII (Sly Syndrome). (2022/12/22) ♡
• Drug Repositioning Applied to Cardiovascular Disease in Mucopolysaccharidosis. (2022/12/12) ♡
• First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B. (2022/12/07) ♡
• 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. Staged Bilateral Total Hip Arthroplasty in a 17-Year-Old With Type VI Mucopolysaccharidosis. (2022/12/06) ♡
• Modeling cartilage pathology in mucopolysaccharidosis VI using iPSCs reveals early dysregulation of chondrogenic and metabolic gene expression. (2022/12/06) ♡
• Flaxseed extract reduces tissue accumulation and enhances urinary excretion of chondroitin sulphate in the rat: a possible new path in substrate reduction therapy for mucopolysaccharidosis. (2022/12/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Juvenile mucopolysaccharidosis plus disease caused by a missense mutation in VPS33A. (2022/12/01) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells. (2022/12/01) ♡
• Diagnosis of patients with mucopolysaccharidosis type II via RNA sequencing. (2022/12/01) ♡

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