Following our request to receive important and timely updates on their SMA clinical programme, Roche has informed us that worldwide recruitment has completed for the RAINBOWFISH clinical trial, which investigates the efficacy and safety of risdiplam in infants with pre-symptomatic SMA Type 1. This milestone marks the enrolment of 26 participants from across the globe.

Following our request for regular updates, Roche has shared an overview of their work in the community over the past year and a summary of progress in advancing research and increasing access to risdiplam in Europe and beyond.

Scholar Rock has announced the design of SAPPHIRE, a Phase 3 clinical trial to further evaluate the safety and efficacy of apitegromab in those living with SMA. The SAPPHIRE study is part of the company’s ongoing development programme for apitegromab.

In response to our request, Roche is pleased to inform us about the initiation of MANATEE, a new global Phase 2/3 clinical study to evaluate the safety and efficacy of GYM329 (RO7204239), an investigational anti-myostatin antibody targeting muscle growth in combination with risdiplam in SMA.