Informed by patient experience, guided by expertise in outcome measure design, regulatory strategy, and decentralized trial conduct.
Christine McSherry founded Jett Foundation in 2001, when her son now 24-year-old Jett was diagnosed with Duchenne Muscular Dystrophy. As head of the Jett Foundation, Christine co-founded the International Duchenne Alliance, a partnership of over 40 independent organizations that has funded nearly $15 million dollars in research to date.
Christine has advocated for the patient perspective to be considered by the FDA when reviewing safe and potentially effective treatments for Duchenne. In April of 2016, Christine presented the Jett Foundation's Patient Centered Outcomes report at the FDA's meeting of the Peripheral and Central Nervous System Drugs Advisory Committee.
Drawing upon her background in project management, software development and information architecture, Mindy Leffler 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.
Director of Outcome Measure Development
Director of Operations
Director of Quality
Clinical Operations Consultant
Director of Finance
Director of Research