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MPS - Mucopolysaccharidosis clinical trials

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NCT ID: NCT03632213 Active, not recruiting - Clinical trials for Mucopolysaccharidosis VI

Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI

Start date: November 7, 2018
Phase: Phase 2
Study type: Interventional

Mucopolysaccharidoses (MPS) are multisystemic diseases with significant clinical overlap between their types, with cardiac problems being among the most commonly observed manifestations and are also among the main causes of mortality in these patients. For some of the cardiovascular manifestations, such as aortic root dilation and valve diseases, there is no effective treatment currently available. Losartan, on the other hand, has been shown to be an effective drug for dilation of the aortic root, at least in animal models. This study aims to evaluate the safety and efficacy of losartan in patients with MPS VI and other mucopolysaccharidoses.

NCT ID: NCT03150069 Completed - Morquio Disease Clinical Trials

Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?

MorqPreg
Start date: May 10, 2017
Phase:
Study type: Observational

The present study seeks to interview women with Morquio A and Morquio B syndrome, to explore their concerns surrounding pregnancy and the impact of ERT on their perspectives, in comparison with the control group of Morquio B subjects for whom no ERT treatment exists. Interviews will be conducted by a health psychologist, in-person or over the telephone. Data will be analyzed using MAXQDA 12.0 software and Grounded Theory. Differences in thematic trends between Morquio A subjects, for whom treatment exists, and a control group of Morquio B subjects, for whom there is no treatment, will be compared.