P230 Safety update: Risdiplam clinical trial program for spinal muscular atrophy (SMA)

Risdiplam (EVRYSDI ®) is a centrally and peripherally distributed, oral survival of motor neuron 2 (SMN2) pre‑mRNA splicing modifier approved for the treatment of SMA in over 90 countries worldwide. The risdiplam clinical development program consists of four clinical trials: 1) FIREFISH (NCT02913482) in infants wit h Type 1 SMA (inclusion criteria [IC]: aged 1–7 months at enrollment). 2) SUNFISH (NCT02908685) in patients with Types 2/3 SMA (IC: aged 2–25 years at enrollment). 3) JEWELFISH (NCT03032172) in patients with SMA (IC: aged 6 months–60 years at enrollment) who previously received RG7800 (RO68852 47), nusinersen (SPINRAZA®), olesoxime or onasemnogene abeparvovec (ZOLGENSMA®).

Source: Neuromuscular Disorders - October 1, 2023 Category: Neurology Authors: G. Baranello, C. Chiriboga, L. Servais, B. Darras, J. Day, N. Deconinck, M. Farrar, R. Finkel, E. Bertini, J. Kirschner, M. Rasson, M. Mazurkiewicz-Be łdzińska, D. Vlodavets, S. Bader-Weder, K. Gorni, B. Jaber, W. Yeung, G. Papp, R. Scalco, E. Mercuri, Source Type: research