EL-PFDD Meeting

Amplifying the Patient Voice

Cure LBSL hosted an externally-led Patient-Focused Drug Development (EL-PFDD) meeting on August 1, 2025, bringing together members of the LBSL community to share their lived experiences, challenges, and hopes for future treatments with regulators, researchers, and drug developers. Explore the meeting and hear directly from the LBSL community about what matters most as we work toward a treatment.