New results from the SUNFISH and JEWELFISH clinical trials with risdiplam in SMA

In two publications released in February 2023, the results of two clinical trials evaluating risdiplam (Evrysdi) in SMA type II and III (SUNFISH trial) and in participants previously treated with nusinersen (Spinraza), onasemnogene abeparvovec (Zolgensma), or olesoxime (JEWELFISH trial) show:

• for the SUNFISH trial: gains in motor function achieved after 12 months of treatment were maintained or improved after 24 months of treatment.
• for the JEWELFISH trial: after one year, the treatment was well tolerated by the 174 participants.

 

Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA). Oskoui M, Day JW, Deconinck N et al. J Neurol. 2023 Feb.

 

Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study. Chiriboga CA, Bruno C, Duong T et al. Neurol Ther. 2023 Feb.