Risdiplam▼: pharmacokinetic, pharmacodynamic, safety and efficacy exposure response analyses ​

FIREFISH (NCT02913482) is assessing the safety, tolerability, PK/PD and efficacy of risdiplam▼ in infants with Type 1 SMA aged 1–7 months. SUNFISH (NCT02908685) is assessing the safety, tolerability, PK/PD and efficacy of risdiplam▼ in patients with Type 2 or 3 SMA aged 2–25 years. JEWELFISH (NCT03032172) is assessing the safety, tolerability and PK/PD of risdiplamv in in non-naïve patients with SMA, aged 6 months to 60 years. This poster presents PK and PD data obtained from all risdiplam▼ clinical studies in patients with SMA, from infants to adults (FIREFISH Parts 1 and 2, SUNFISH Parts 1 and 2 and JEWELFISH). This analysis will add to the understanding of the PK/PD profile of risdiplam▼ in individuals with SMA.