Table 2.
Demographic characteristics and SMA classification of adults treated with risdiplam in the studied cohorts
| Study (year, country) | N | Age (years) | SMA type 1 n/N (%) | SMA type 2 n/N (%) | SMA type 3 n/N (%) | SMA type 4 n/N (%) | Ambulatory n/N (%) | Non-ambulatory n/N (%) |
|---|---|---|---|---|---|---|---|---|
| Mercuri et al., 2022 (International) [4] | 14 | NR (range 18–25) | 0/14 (0%) | NR | NR | 0/14 (0%) | NR | NR |
| Oskoui et al., 2023 (International) [11] | 10 | NR (range 18–25) | 0/10 (0%) | NR | NR | 0/10 (0%) | NR | NR |
| Keritam et al., 2025 (Austria) [12] | 57° | 35.7 (28.8–43.4)a | 2/57 (3.5%) | 23/57 (40.4%) | 27/57 (47.4%) | 4/57 (7.0%) | 13/57 (22.8%) | 44/57 (77.2%) |
| Iterbeke et al., 2025 (Belgium) [11] | 18 | 38.6 ± 13.5b | 0/18 (0%) | 8/18 (44.4%) | 9/18 (50.0%) | 1/18 (5.6%) | 2/18 (11.0%) | 16/18 (89%) |
| Belančić et al., 2024 (Croatia) [14] | 7^ | 35.0 (21.25–44.5)ᵇ | 0/7 (0%) | 0/7 (0%) | 7/7 (100%) | 0/7 (0%) | 7/7 (100%) | 0/7 (0%) |
| Gavriilaki et al., 2025 (Greece) [15] | 14 | 30 (18–57)ᶜ | 0/14 (0%) | 11/14 (78.6%) | 3/14 (21.4%) | 0/14 (0%) | 1/14 (7.1%) | 13/14 (92.9%) |
| Kessler et al., 2024 (Germany) [16] | 18 | 37.8 ± 3.2b | 0/0 (0%) | 7/18 (38.9%) | 11/18 (61.1%) | 0/0 (0%) | 2/18 (11.1%) | 16/18 (88.9%) |
| Bjelica et al., 2024 (Germany) [17] | 14* | 34.5 (18–51)ᶜ | 0/14 (0%) | 8/14 (57.1%) | 6/14 (42.9%) | 0/14 (0%) | 1/14 (7.1%) | 13/14 (92.9%) |
| Sitas et al., 2024 (Croatia) [18] | 31 | 30 (18–65)ᶜ | 0/31 (0%) | 15/31 (48.4%) | 16/31 (51.6%) | 0/31 (0%) | 6/31 (19.4%) | 25/31 (80.7%) |
| Ñungo Garzón et al., 2023 (Spain) [19] | 6 | 35.5 (17–46)ᶜ | 0/6 (0%) | 6/6 (100%) | 0/6 (0%) | 0/6 (0%) | 0/6 (0%) | 6/6 (100%) |
| Severa et al., 2024 (France) [20] | 6 | 41 (21–65)ᶜ | 0/6 (0%) | 4/6 (66.7%) | 2/6 (33.3%) | 0/6 (0%) | 0/6 (0%) | 6/6 (100%) |
| Brakemeier et al., 2024 (Germany) [21] | 25 | 34.3 ± 11.3b | 0/25 (0%) | 24/25 (96.0%) | 1/25 (4.0%) | 0/25 (0%) | 0/25 (0%) | 25/25 (100%) |
| McCluskey et al., 2023 (N. Ireland) [22] | 6 | 33.7 (26–44)ᶜ | 0/6 (0%) | 6/6 (100%) | 0/6 (0%) | 0/6 (0%) | 0/6 (0%) | 6/6 (100%) |
| Schön et al., 2022 (Portugal) [24] | 1 | 27 | 0/1 (0%) | 1/1 (100%) | 0/1 (0%) | 0/1 (0%) | 0/1 (0%) | 1/1 (100%) |
Overview of demographic and clinical characteristics of adult patients with SMA treated with risdiplam throughout different studies. Data include: title and country of study, sample size (N), age, SMA type distribution, and ambulatory status. Unless otherwise specified, data are presented as number (n) and percentage (%). °Keritam included 1 patient out of 57 with unknown SMA type. ^Belančić described a mixed pediatric and adult cohort, being the 7 patients with SMA type 3a (“a” as “adult”, “p” as “pediatric”) the adult-only one. *Bjelica baseline N = 14 (8 SMA2/6 SMA3); motor outcomes (RULM/HFMSE) evaluable patients vary by timepoint, with RULM classification detailed on 13 patients. aMedian (IQR); bMean ± SD; cMedian (range). Abbreviations: SMA = Spinal Muscular Atrophy