G12
Conditions
Interventions
Group 1: One Arm observational study in the overall population of SMA Patients in the German register if SMA patients
Sponsors
Initiative „Forschung und Therapie für die Spinale Muskelatrophie“ im Förderverein für die Deutsche Gesellschaft für Muskelkranke e.V
Eligibility
Sex/Gender
All
Age
4 Years to No maximum
Inclusion criteria
Inclusion criteria: adequate understanding of German language primary residence in Germany, diagnosed and genetically confirmed spinal muscular atrophy
Exclusion criteria
Exclusion criteria: primary residence any other country than Germany, no diagnosed spinal muscular atrophy, no registration in the contacting SMA register
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The recording of HRQoL and psychopathology (SDQ) based on the total, profile and/or index scores of the questionnaires. Additional recording of socio-economic and clinical parameters (e.g. age, SMA type, current motor skills, school attendance) | — |
Secondary
| Measure | Time frame |
|---|---|
| Review of applicability and psychometric quality criteria and validation of the FB in a clinical sample of patients with SMA | — |
Countries
Germany
Contacts
Public ContactMarina Flotats-Bastardas
Klinik für Allgemeine Pädiatrie und NeonatologieKliniken für Kinder- und JugendmedizinNeuropädiatrieUniversitätsklinikum des Saarlandes
Outcome results
None listed