About Us

Cure LBSL

Cure LBSL is a nonprofit dedicated to raising awareness of LBSL, funding research and supporting affected individuals and their families around the world. Cure LBSL strives to ensure our patients’ voices are heard by regulators, drugmakers and researchers.

Our Mission

To accelerate research, drive the development of effective treatments and a cure for LBSL, while empowering patients and families through education, advocacy and a supportive global community.

Our Vision

A world where every individual diagnosed with LBSL has access to life-changing therapies and a cure, allowing them to live full, healthy and unimpeded lives.

Our Accomplishments

  • Raised over $6 million for research

  • Hosted an Externally-Led Patient Focused Drug Development (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 five biennial LBSL conferences

  • Created a global network and helped launch natural history studies in the U.S., Finland and the Netherlands

Giving Hope to Families

A girl playing in fallen leaves

“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)

Two boys in plaid shirts

“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)

A boy with LBSL sitting outdoors

“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)

Advocacy Spotlight

LBSL Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting

Cure LBSL hosted this meeting on Aug. 1, 2025, in Washington, D.C., for the LBSL community to share their experiences and hopes for a future treatment with regulators, researchers and drug developers. A Voice of the Patient report is forthcoming.