Patients & Families
You inspire everything we do.
Living with a severe genetic disease affects the whole family. Explore our clinical studies and the ways we're partnering with the community.
Clinical Studies
Our ASCEND Phase 3 study is now underway. Participation criteria, study sites, and family resources are available through our clinical operations team.
- → Global multi-center trial across North America, Europe, Japan
- → Open-label extension for eligible participants
- → Dedicated family concierge support