The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.

  1. Roche completes recruitment for JEWELFISH clinical trial

    Roche completes recruitment for JEWELFISH clinical trialRoche 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.

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