Rare disease.
Real hope.
We are closer than ever to a gene therapy for LBSL. Help us get there!
Meet Some of Our Motivation
We’re on a mission to find a cure for LBSL, raise awareness, and support patients and families. These individuals are just part of our reason why!
The LBSL externally-led Patient-Focused Drug Development meeting on Aug. 1 provided our community with a critical opportunity to share their lived experiences and hopes for a treatment. We are developing the Voice of the Patient report to summarize the meeting and capture insights from the LBSL community to help inform future drug development efforts and will publish it in the coming months!