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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)

• Evaluation of aortic elasticity properties in mucopolysaccharidosis patients; effect of enzyme replacement therapy (ERT) on aortic stiffness. (2024/06/27) ♡
• Capsular and retinaculum thickening in type II mucopolysaccharidosis: a novel MRI finding. (2024/06/01) ♡
• Clinical and Molecular Characterization of Mucopolysaccharidosis Type 3A and 3B in a Turkish Series. (2024/06/01) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Corrigendum to "Enhanced osteoblastic differentiation of parietal bone in a novel murine model of mucopolysaccharidosis type II" [Molecular Genetics and Metabolism Reports Vol. 37, December 2023, 101021]. (2024/05/28) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Late-onset mucopolysaccharidosis type IIIA mimicking Usher syndrome. (2024/05/01) ♡
• 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. Ocular manifestations of liver disease: an important diagnostic aid. (2024/04/15) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Expanding the phenotypic and genotypic spectrum of patients with HGSNAT-related retinopathy. (2024/04/01) ♡
• Training of community health agents - a strategy for earlier recognition of mucopolysaccharidoses. (2024/04/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Bow Hunter Syndrome: An Illustrative Case and Operative Management. (2024/02/01) ♡
• Natural history of cardiac findings in mucopolysaccharidosis type I: report from an international registry. (2024/02/01) ♡
• Ability change across multiple domains in mucopolysaccharidosis (Sanfilippo syndrome) type IIIA. (2024/02/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Evaluation of etanercept (a tumor necrosis factor alpha inhibitor) as an effective treatment for joint disease in mucopolysaccharidosis type I. A case report with whole-body magnetic resonance imaging. (2024/01/19) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Anesthesia management in a child with mucopolysaccharidosis and toxic epidermal necrolysis: A case report. (2024/01/01) ♡
• Adenotonsillar pathology in mucopolysaccharidoses - lysosomal storage predominates in paracortical CD63+ cells. (2024/01/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Bilateral Hunter's bow syndrome: a rare case diagnosed by dynamic digital subtraction angiography. (2024/01/01) ♡
• Evaluation of cardiac findings in mucopolysaccharidosis. (2024/01/01) ♡
• Glycosaminoglycan-induced proinflammatory cytokine levels as disease marker in mucopolysaccharidosis. (2024/01/01) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Repetitive, non-invasive imaging of neurodegeneration, and prevention of it with gene replacement, in mice with Sanfilippo syndrome. (2024/01/01) ♡
• The importance of skeletal x-ray screening for dysostosis multiplex in the early diagnosis of mucopolysaccharidosis. (2024/01/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Clinical letter new-onset epilepsy presenting as non-convulsive status epilepticus in Mucopolysaccharidosis type II: A case report. (2024/01/01) ♡
• Study of the peripheral and central auditory pathways in patients with mucopolysaccharidosis. (2024/01/01) ♡
• Tandem mass spectrometric assay of N-acetylglucosamine-6-sulfatase for multiplex analysis of mucopolysaccharidosis-IIID in dried blood spots. (2024/01/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Use of Marsupialization as a Definitive Treatment for Large-sized Dentigerous Cysts in a Patient with Mucopolysaccharidosis Type I. (2024/01/01) ♡
• Lysosomal storage diseases. (2024/01/01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. CO2 Supplement for Treatment of Acute Mountain Sickness (2024-12-18) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Gene Therapy in Patients With Mucopolysaccharidosis Disease (2024-11-22) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Effect of Chronic Feeding of Inulin And Methylcellulose on Colonic Fermentation (2024-11-15) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products (2024-10-30) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Hunter Outcome Survey (HOS) (2024-10-28) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Open-label Study of Anakinra in MPS III (2024-10-22) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Longitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI (2024-10-22) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS II (2024-10-15) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. MPS II Immunophenotyping (2024-09-19) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Sevoflurane Wash in Method Using Conventional Flows Versus Low Flows of 0.5L (2024-08-27) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients (2024-08-06) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I (2024-07-25) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers (2024-07-03) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome (2024-06-10) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX (2024-05-03) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Effect of Rate Of Delivery on Colonic Fermentation of inuliN (EON) (2024-05-01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study of GC1111 in Hunter Syndrome Patients (2024-04-16) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases (2024-03-25) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3) (2024-03-12) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) (2024-03-05) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants (2024-02-20) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia (2024-02-15) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting (2024-02-14) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia (2024-02-08) ♡
• Altered Sphingolipid Hydrolase Activities and Alpha-Synuclein Level in Late-Onset Schizophrenia. (2023/12/31) ♡
• Molecular docking analysis of a dermatan sulfate tetra-saccharide to human alpha-L-iduronidase. (2023/12/31) ♡

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