ongitudinal Data Collection from Patients with Spinal Muscular Atrophy: The SMArtCARE Database
Recruiting
- Conditions
- G12.0Infantile spinal muscular atrophy, type I [Werdnig-Hoffman]
- Registration Number
- DRKS00012699
- Lead Sponsor
- niversitätsklinikum Freiburg; Klinik für Neuropädiatrie und Muskelerkrankungen
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 2500
Inclusion Criteria
• All patients with genetically confirmed 5q SMA with residence in German-speaking regions.
• Informed consent provided by patient/caregiver
Exclusion Criteria
• Further types of SMA (non 5q SMA)
• Participation in an AMG trial
Study & Design
- Study Type
- observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method The aim of SMArtCARE is to collect data from all SMA patients during routine patient visits. SMArtCARE collects data on motor function as well as changes in respiratory function, nutritional status and orthopedic symptoms. To evaluate changes in motor function, standardized physiotherapeutic assessments are performed.
- Secondary Outcome Measures
Name Time Method