Roche Completes Recruitment for JEWELFISH Clinical Trial

28 February 2020

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 (Zolgensma).

RAINBOWFISH is now the only global clinical trial with 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.

Further information:

The community update
About risdiplam
The RAINBOWFISH trial
Roche’s SMA website