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 announces new clinical trial for RG7916 in spinal muscular atrophy

    Roche, along with partners PTC Therapeutics and the SMA Foundation in the US, recently provided an update on the development of RG7800 and announced a new clinical trial planned for RG7916. The study will take place in The Netherlands.

    RG7916 is another SMN2 splicing modifier. This new investigational medicine will now advance to its very first clinical study in healthy individuals in January 2016, which will provide important information about the safety profile, pharmacokinetics (what the body does to the medicine) and effects of RG7916 in healthy individuals.

    RG7916 has different characteristics relative to RG7800, which may influence how the medicine interacts with the body and this will be evaluated in the study.

    The clinical study or RG7800, or Moonfish, in people with SMA, was placed on clinical hold. This was a precautionary measure after an unexpected safety finding was identified in an animal study exploring long term treatment with RG7800 at exposures above those observed in the Moonfish study. The Moonfish study remains on hold as we continue investigations to understand these findings.

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