Mindy Leffler is the Managing Director of the Qualitative Research and Psychometrics group at Emmes Endpoint Solutions (formerly Casimir). Drawing upon her background in project management, software development, and information architecture, she designed a PRO program for her son, Aidan, which proved instrumental in the FDA approval of eteplirsen, the first FDA-approved treatment for Duchenne. Mindy has used her experience as a member of the patient and caregiver community to inform an approach that engages patients, KOLs, and regulators in order to develop more sensitive and meaningful endpoints in a range of neuromuscular and neurodevelopmental disorders.