NIEMANN-PICK

The Niemann-Pick Type C (NPC) externally led Patient-Focused Drug Development (PFDD) meeting is a groundbreaking initiative that will give persons with NPC disease and their advocates the opportunity to provide the Food and Drug Administration (FDA), drug developers, and NPC stakeholders with perspectives from our community on a number of important issues... The meeting followed the successful model developed by the FDA for similar Agency-convened meetings and provide persons with NPC the unique opportunity to share their story and experiences-including the challenges they face on a daily basis in living with NPC and their hopes for future treatments-with senior FDA officials and industry to better inform how therapies are developed and how the agency evaluates and considers potential therapies for the disease... The Ara Parseghian Medical Research Fund isdedicated to funding medical research projects to find a treatment for Niemann-Pick Type C (NP-C) disease.

Primary location: Notre Dame United States