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posted on 19.01.2022, 14:18 by Valerie Aponte Ribero, Monica Daigl, Yasmina Martí, Ksenija Gorni, Rachel Evans, David Alexander Scott, Anadi Mahajan, Keith R Abrams, Neil Hawkins

- Statistical Analysis Plan

- Supplementary Figure 1. PRISMA flow chart of the systematic literature review.

- Supplementary Figure 2. Evidence networks in Type 1 and Types 2/3 SMA.

- Supplementary Figure 3. Histogram of re-scaled weights in the ENDEAR-matched FIREFISH population (total N=58).

- Supplementary Figure 4. Overall survival in patients with Type 1 SMA treated with nusinersen, risdiplam and BSC.

- Supplementary Figure 5. Histograms of re-scaled weights in the CHERISH-matched SUNFISH population.

- Supplementary Figure 6. Matching of SUNFISH and CHERISH placebo groups for RULM and HFMSE.

- Supplementary Table 1. PICOS framework and additional search criteria for the SLR.

- Supplementary Table 2. List of data sources used in the SLR.

- Supplementary Table 3. Key deviations from statistical analysis plan and justification.

- Supplementary Table 4. Clinical trials excluded from the SLR.

- Supplementary Table 5. Availability of endpoints of interest in Type 1 SMA.

- Supplementary Table 6. Baseline characteristics of FIREFISH pre- and post‑matching with STR1VE‑US (MAIC analysis).

- Supplementary Table 7. STC model fit statistics of FIREFISH versus STR1VE-US.

- Supplementary Table 8. BSID-III covariate estimates (STC FIREFISH vs STR1VE-US).

- Supplementary Table 9. CHOP-INTEND covariate estimates (STC FIREFISH vs STR1VE-US).

- Supplementary Table 10. Availability of endpoints of interest in Types 2 and 3 SMA.

- Supplementary Table 11. Analyses of HFMSE outcomes in SUNFISH and CHERISH at Month 12.


Funded by F. Hoffmann-La Roche Ltd