Patients
risdiplam
A total of 180 patients were enrolled in SUNFISH Part 2 and were randomized to receive risdiplam (After month 12, all patients received risdiplam. A total of 176 patients entered the open-label treatment period (defined as months 12–24 in the study). Motor function scores from the 59 patients who switched from placebo to risdiplam at study month 12 (i.e., at adjusted baseline) are presented in Table S1. Patients and all individuals in direct contact with patients at the site remained blinded to the treatment group from randomization until completion of at least their second year in the study.During the open-label treatment period, two patients withdrew from treatment. One of these patients elected to withdraw from the study but completed the month 24 study visit. The other patient withdrew prematurely due to the COVID-19 pandemic and did not complete the month 24 visit. Data are available from 164 patients who were recorded as having completed the open-label treatment period at month 24 by the clinical cut-off date of 30th September 2020 (10 patients did not have a recorded date of completion for the open-label treatment period). The clinical cut-off date is the date at which it was estimated that the last patient in Part 2 would have completed the month 24 study visit. However, some patients missed this study visit due to COVID-19 pandemic restrictions. All patients remaining in the study entered the open‑label extension phase (≥ 24 months) for 3 years of further treatment.