Today, new data was presented demonstrating the ongoing benefit of risdiplam (RG7916) for the treatment of all types of spinal muscular atrophy (SMA). The data was presented at the 24th International Annual Congress of the World Muscle Society. Presentations include data from the FIREFISH, SUNFISH, and JEWELFISH clinical trials. The SMA program is a collaboration […]

AveXis, a Novartis company, recently announced that new interim data from the Phase 3 SPR1NT trial in pre-symptomatic patients as well as interim data from the ongoing Phase 3 STR1VE clinical program for Zolgensma showed positive outcomes, demonstrating age‑appropriate major milestone gain with pre‑symptomatic treatment and prolonged event-free survival* in patients with SMA Type 1.

The annual SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year, we had a record setting 735 attendees join together in Anaheim, CA. The goal of the meeting is to create open communication of early, unpublished data, accelerating the pace of research. The meeting also furthers research