Validation and Reliability of Iris Cameras in Mucopolysaccharidoses
Condition:   Mucopolysaccharidoses Intervention:   Other: Imaging Sponsors:   Manchester University NHS Foundation Trust;   Orchard Therapeutics Not yet recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - April 29, 2022 Category: Research Source Type: clinical trials

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
(Source: Trials)
Source: Trials - December 9, 2021 Category: Research Source Type: clinical trials

Establishing a core outcome set for mucopolysaccharidoses (MPS) in children: study protocol for a rapid literature review, candidate outcomes survey, and Delphi surveys
DiscussionThe proposed COS will inform the choice of outcomes to include in the MPS registry and, more broadly, promote the standardized collection of patient-oriented outcomes for pediatric MPS research. By involving patients/families from the earliest stage of the research, we will ensure that the COS will be relevant to those who will ultimately benefit from the research.Trial registrationPROSPEROCRD42021267531, COMET (Source: Trials)
Source: Trials - November 17, 2021 Category: Research Source Type: clinical trials

Cardiovascular Structure and Function in the Mucopolysaccharidoses
Conditions:   Mucopolysaccharidoses;   Carotid Disease;   Cardiac Disease;   Inflammation Interventions:   Diagnostic Test: Carotid ultrasonography;   Diagnostic Test: Echocardiography, transthoracic;   Procedure: Venipuncture Sponsors:   Children's Hospital of Orange County;   Ultragenyx Pharmaceutical Inc Recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - October 1, 2021 Category: Research Source Type: clinical trials