VKS/Erasmus MC Mucopolysaccharidosis Survey

Recruiting

• Conditions: 10021605; Hurler's syndrome; Hunter's syndrome; Sanfilippo's syndrome; 10028396; Maroteaux-Lamy's syndrome; Morquio's syndrome; mucopolysaccharidosis; Sly's syndrome; 10027424

• Registration Number: NL-OMON35162
• Lead Sponsor: Erasmus MC, Universitair Medisch Centrum Rotterdam

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 6 May 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: Not specified
• Target Recruitment: 100

Inclusion Criteria

All patients (all ages) who have signed the VKS / Mucopolysaccharidosis Survey patient information and authorization form and have a confirmed diagnosis of mucopolysaccharidosis are eligible for inclusion.

Exclusion Criteria

none

Study & Design

• Study Type: Observational non invasive
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The results of the Mucopolysaccharidosis Questionnaire will be used to describe<br /><br>the natural course of MPS, the current clinical outcome, the health damage at<br /><br>diagnosis and the effect of treatment. The scores obtained from the<br /><br>standardized questionnaires will be used to measure the consequences of MPS<br /><br>disease on daily activities, quality of life, development and pain and to<br /><br>evaluate. the effects of treatment options. </p><br>

Secondary Outcome Measures

Name	Time	Method
<p>not applicable</p><br>