Our Impact
Cure LBSL is a registered nonprofit dedicated to raising awareness of LBSL, funding research and supporting affected individuals and their families around the world. As the only patient advocacy group in the world focusing on LBSL, Cure LBSL has worked hard to amplify the voices of our patients to regulators, drug makers and researchers. We attend and participate in conferences and symposiums throughout the year.
Annual Reports
Click below to read our annual reports!
Giving Hope to Families
“Your donations transformed LBSL from a rare disease with no cure in sight, to a rare disease with an entire medical team working on a cure. They helped connect me and my family to others affected by this condition. Your support has given our family hope.”
– Hazel’s Mom (Wisconsin)
“A Cure for Ellie (now Cure LBSL) was the first source of help we found after our family received the LBSL diagnosis. Ellie and her family gave us the strength and hope we desperately needed when everyone else was telling us there was none. We are forever grateful and blessed to have found Ellie and her family.”
– Dean & Dalton’s Mom (Missouri)
“Suffering from such a rare disease means finding a drug therapy will never be a priority for the pharmaceutical industry and it may never come in time…but because of your support for this foundation, we now have a voice and the chance to find a breakthrough therapy.”
– Tasso’s Mom (Greece)
Our Advocacy
LBSL Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting
Cure LBSL hosted this hybrid impactful meeting on Aug. 1, 2025, in Washington D.C. as a platform for our LBSL community to share with their lived experiences and hopes for a future treatment with the U.S. Food and Drug Administration and other regulators, researchers and drug developers. The Voice of the Patient report to summarize the event is being written and will be posted on our website as well as the FDA’s once finalized.
LBSL Patient Listening Session
Cure LBSL co-hosted a Patient Listening Session with the U.S. Food and Drug Administration on May 23, 2023, to share the patient voice with regulators, reviewers and others who will be involved in the evaluation and approval process for future treatments. During the 90-minute closed-door virtual meeting, nine patients shared what it’s really like to live with LBSL, in their own words or through their caregivers. Want to review of copy of the report or other meeting materials? Email info@curelbsl.org.