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. Cytokinetics’ reldesemtiv shows encouraging results in adolescent & adult SMA patients

    Cytokinetics' reldesemtiv shows encouraging results in adolescent & adult SMA patientsCytokinetics presented encouraging results from the Phase 2 clinical study of reldesemtiv in patients with SMA at the Cure SMA Researchers’ conference in Dallas.

    What is reldesemtiv and what is it meant to do?

    Reldesemtiv is a fast skeletal muscle troponin activator (FSTA) which slows the rate of calcium release from the regulatory troponin complex of fast skeletal muscle fibers. This means that this compound can increase the force generated by skeletal muscle and delays the onset and extent of muscle fatigue. The hypothesis is that reldesemtiv might improve muscle function and physical performance in people with SMA.

    What do the phase 2 clinical trial results show?

    The study showed dose- and concentration-dependent increases in time to muscle fatigue as measured by changes from baseline in the Six Minute Walk Distance (6MWD), a sub-maximal exercise test of aerobic capacity and endurance and Maximal Expiratory Pressure (MEP), a measure of strength of respiratory muscles, after eight weeks of treatment with reldesemtiv.

    This study provides the first data indicating that a muscle-directed therapy, namely a fast skeletal muscle troponin activator, may be clinically beneficial in patients with SMA. This data is especially encouraging given the unmet need among those adolescent and adult individuals with SMA who have persistent muscle weakness, fatigue and functional impairment.

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