At last week’s Annual SMA Conference, Cytokinetics released additional data from their Phase 1 study of CK-2127107, a skeletal muscle activator. Cytokinetics, in partnership with Astellas, has already announced that a Phase 2 study will begin later this year. Our partnership with Cytokinetics highlights two strengths of Cure SMA’s research model: providing seed funding to […]

Isis Pharmaceuticals provided an update on its ongoing open-label Phase 2 clinical study of ISIS-SMNRx in infants with Type I spinal muscular atrophy (SMA). Previously the company reported data from this study on event-free survival, measures of muscle function and assessments of developmental milestones. The data reported now shows continued increases in median event-free survival