Health Related Quality of life in Patients with SMA

• Conditions: G12; Spinal muscular atrophy and related syndromes

• Registration Number: DRKS00022876
• Lead Sponsor: Initiative „Forschung und Therapie für die Spinale Muskelatrophie im Förderverein für die Deutsche Gesellschaft für Muskelkranke e.V

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2020-10-19
• Study Completion: 2021-09-05
• Last Update Posted: 19 August 2024

Recruitment & Eligibility

• Status: Complete
• Sex: All
• Target Recruitment: 113

Inclusion Criteria

adequate understanding of German language
primary residence in Germany, diagnosed and genetically confirmed spinal muscular atrophy

Exclusion Criteria

primary residence any other country than Germany, no diagnosed spinal muscular atrophy, no registration in the contacting SMA register

Study & Design

• Study Type: observational
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
The recording of HRQoL and psychopathology (SDQ) based on the total, profile and/or index scores of the questionnaires.<br>Additional recording of socio-economic and clinical parameters (e.g. age, SMA type, current motor skills, school attendance)

Secondary Outcome Measures

Name	Time	Method
Review of applicability and psychometric quality criteria and validation of the FB in a clinical sample of patients with SMA