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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)

• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Bow Hunter's Syndrome. (2022/01/01) ♡
• Corrigendum to "Comparative analysis of brain pathology in heparan sulphate storing mucopolysaccharidosis" [Molecular Genetics and Metabolism 131 (2020) pages 197-205]. (2022/01/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. [The combination of recurrent otitis media and adenotomy in early childhood as diagnostic marker of mucopolysaccharidosis type II (Hunter syndrome)]. (2022/01/01) ♡
• [Application of genome editing technology in gene therapy]. (2022/01/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Delayed Onset Post-Operative Neurologic Deficit in a Patient With Mucopolysaccharidosis type VI: A Case Report. (2022/01/01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI (2022-12-19) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study of JR-141 in Patients With Mucopolysaccharidosis II (2022-11-14) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study of JR-141 in Patients With Mucopolysaccharidosis Type II (2022-11-14) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB) (2022-10-26) ♡
• 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-913 in Subjects With MPS II (2022-10-25) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Human Placental-Derived Stem Cell Transplantation (2022-10-25) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Assessment and Validation of Electronic Gas and Bloating Diary (2022-10-24) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Mucopolysaccharidosis Type II Observational (2022-10-10) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101 (2022-05-31) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study to Improve the Awareness of Mucopolysaccharidosis Type II in Brazil (2022-05-09) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB (2022-05-05) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Mucopolysaccharidosis Type II Natural History (2022-04-26) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. The Intensively Follow-up Examinations for Asymptomatic MPS I Infants in Taiwan (2022-03-17) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. The Effect of Paula Method Exercises on Post-cesarean Section Distension (2022-03-17) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. End Tidal Anesthetic Concentration in Different Anesthesia Techniques Where Depth of Anesthesia Adjusted With Entropy (2022-03-17) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients (2022-02-25) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Effects of Resistant Potato Starch on the Gut Microbiota (2022-02-16) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Awareness in Old Aged Patients During Laryngoscopy and Intubation Using Isolated Forearm Technique (2022-01-13) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Respiratory Cathepsins, Proteases Inhibitors and Glycosaminoglycans (GAG) in Mucopolysaccharidosis (2022-01-04) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Drosophila D-idua Reduction Mimics Mucopolysaccharidosis Type I Disease-Related Phenotypes. (2021/12/31) ♡
• Mucopolysaccharidosis Type II: A Kenyan Case Series. (2021/12/22) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. A fortuitous but characteristic blood smear observation allowing a late diagnosis of MPS-VII. (2021/12/17) ♡
• COVID-19 impact on the diagnosis of Inborn Errors of Metabolism: Data from a reference center in Brazil. (2021/12/17) ♡
• 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. Damage of the cardiovascular system in lysosomal storage disease - mucopolysaccharidosis. (2021/12/16) ♡
• 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 Type VI, an Updated Overview of the Disease. (2021/12/15) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Mucopolysaccharidosis Type I Diagnosed by Aortic and Mitral Valve Replacement. (2021/12/15) ♡
• Correction to: Establishing a core outcome set for mucopolysaccharidoses (MPS) in children: study protocol for a rapid literature review, candidate outcomes survey, and Delphi surveys. (2021/12/09) ♡
• Response to Letter to the Editor: Secondary ganglioside G(M2) accumulation in mucopolysaccharidoses. (2021/12/08) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Analysis of cognitive ability and adaptive behavior assessment tools used in an observational study of patients with mucopolysaccharidosis II. (2021/12/04) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Mucopolysaccharidosis type IV: report of 5 cases of Morquio Syndrome. (2021/12/03) ♡
• Mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome): defining and measuring functional impacts in pediatric patients. (2021/12/02) ♡
• Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. Long-term safety and clinical outcomes of intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A. (2021/12/01) ♡
• Identification and structure characterization of novel IDS variants causing mucopolysaccharidosis type II: A retrospective analysis of 30 Chinese children. (2021/12/01) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Neuropathology of murine Sanfilippo D syndrome. (2021/12/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Bilateral Perthes Masking Morquio Disease. (2021/12/01) ♡
• 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. Bone Biomarkers in Mucopolysaccharidoses. (2021/11/23) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. A 1-year and 4-month-old child with mucopolysaccharidoses type II: A clinical case report from Ethiopia. (2021/11/22) ♡
• Music therapy and Sanfilippo syndrome: an analysis of psychological and physiological variables of three case studies. (2021/11/20) ♡
• Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome. (2021/11/18) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. The Beginning of the End of Allogeneic Transplantation for Hurler Syndrome? (2021/11/18) ♡
• Angle-closure glaucoma with attenuated mucopolysaccharidosis type I in a Chinese family. (2021/11/18) ♡
• Establishing a core outcome set for mucopolysaccharidoses (MPS) in children: study protocol for a rapid literature review, candidate outcomes survey, and Delphi surveys. (2021/11/17) ♡
• The Association Between Lysosomal Storage Disorder Genes and Parkinson's Disease: A Large Cohort Study in Chinese Mainland Population. (2021/11/15) ♡
• 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. Loss of Function of Mutant IDS Due to Endoplasmic Reticulum-Associated Degradation: New Therapeutic Opportunities for Mucopolysaccharidosis Type II. (2021/11/12) ♡
• Reproduction in Animal Models of Lysosomal Storage Diseases: A Scoping Review. (2021/11/12) ♡

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