First patient treated in the ASCEND study evaluating the potential benefit of investigational higher dose nusinersen in children, teens and adults previously treated with Evrysdi® (risdiplam) Baseline characteristics indicate all nine infants and toddlers enrolled in RESPOND had suboptimal clinical status in ≥2 areas after receiving Zolgensma® (onasemnogene abeparvovec); there were no new safety findings with subsequent […]

Cure SMA is pleased to announce the launch of an expanded Phase 8 of our SMA Industry Collaboration. The SMA Industry Collaboration is a multi-faceted partnership that brings together pharmaceutical companies, SMA Europe, Cure SMA, and other nonprofit organizations to share information, ideas, and data to benefit the broader SMA community. Through the Industry Collaboration, we fund

We’d like to give a warm welcome to Children’s Hospital Colorado as a newly added Cure SMA Care Center! Thank you to Loree, Ward and Lyza Weisman for your generous donation supporting your local SMA Care Center – the Children’s Hospital Colorado. We also thank the Weisman Family Foundation and the Ritter Family Foundation. The