Fig. 2.
Disease-specific health-related quality of life (HRQOL) of SMA patients. Disease-specific HRQOL was assessed by PedsQL™© Measurement Model, module for neuromuscular disorders, German version 3.0. Higher scores indicate better HRQOL. HRQOL was either patient self-reported or proxy-reported (a) and subdivided into the disease-specific sections: problems with the neuromuscular disease, communication and family resources (b). Mean scores are presented with standard deviations. Scale with 0 = min. and 100 = max. * indicates significant differences (p < 0.001)