Roche has shared a community update to inform us about recruitment completion for their JEWELFISH clinical trial. This milestone marks the enrolment of 174 participants from across the globe.
JEWELFISH is a clinical trial of the small molecule risdiplam. Risdiplam is a small molecule drug taken orally, which targets and encourages the SMN2 ‘back-up’ gene to produce a greater amount of functional SMN protein. This trial is for people aged 6 months to 60 years, who have previously participated in a trial with SMN2-targeting therapies (nusinersen), or a neuroprotector (olesoxime), or a gene therapy (onasemnogene abeparvovec-xioi – Zolgensma).
RAINBOWFISH is now the only global clinical trial wRoche’s community statement – 27 February 2020ith risdiplam currently recruiting. RAINBOWFISH is designed to explore the effect and safety of risdiplam in newborn babies up to 6 weeks old with a genetic diagnosis of SMA, who have not yet shown symptoms.