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!
Our Accomplishments
Raised over $3.5 million for research
Hosted an EL-PFDD meeting in 2025
Co-hosted an FDA Patient Listening Session in 2023
Chan Zuckerberg Initiative Rare as One Network Cycle 2 grantee
2017 Rare Impact Award recipient
Hosted four biennial LBSL Patient Conferences in Baltimore
Created a global network and helped launch natural history studies in the U.S., Finland and the Netherlands
“To any and everyone trying to help figure this out, thank you. It means more than you will ever know.”
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!