Firefly Fund was proud to partner with the Ara Parseghian Medical Research Fund and other NPC patient-inspired foundations such as Dana’s Angels Research Trust, Hope For Marian, National Niemann-Pick Disease Foundation, Inc., Niemann-Pick Canada, The Hide & Seek Foundation for Lysosomal Disease Research, and Jonathan’s Dream, in a historic community-wide collaboration aimed at giving a national voice to NPC families, patients and caregivers at the U.S. Food and Drug Administration.
The Patient Focused Drug Development (PFDD) meeting was held in Maryland last spring, and the report summarizes that meeting as well as the subsequent benefit vs. risk survey in which community members participated. The final report was given to the FDA in hopes of communicating priorities related to drug development for NPC patients.