Additional file 1: Figure S1. of A conceptual disease model for adult Pompe disease

Analyses based on conceptual model Pompe disease (DOCX 41 kb

La Vigie marocaine

17 novembre 19381938/11/17 (A29,N10202)-1938/11/17

Additional file 1: Table S1. of Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease

Survival: Cox regression estimates. Table S2. Quality of life: regresssion estimates conceptual disease model. Table S3. Costs: regression estimates healthcare costs and productivity costs and input cost parameters. Figure S1. Double loop model structure. (PDF 394 kb

Additional file 4: Figure S3. of Lack of robust satellite cell activation and muscle regeneration during the progression of Pompe disease

Unchanged satellite cell proliferation in Pompe disease. Cryosections from Pompe muscle biopsies were co-stained for Pax7 to mark satellite cells and Ki67 (see Fig.4) using immunofluorescence to assess active proliferation. Nuclei were stained with Hoechst (blue; Fig.4). The figure depicts the quantification of Ki67-positive satellite cells in Pompe patients (red symbols; different symbols per patient group), controls (green symbols), and a DMD patient (blue symbol). Data are expressed as % Ki67-positive satellite cells per total nuclei. Pompe patients: group 1 n = 3, group 2 n = 3, group 3 n = 4, group 4 n = 3. DMD patient: n = 1. Controls: infantile n =1, juvenile n = 2, adult n = 5. There were no statistical differences between patient and control groups. (PDF 963 kb