Enhancing Awareness and Understanding of
Gene Therapy among Rare Disease Communities:
A Research-Driven Roadmap

This study, conducted by Engage and its partner SmithSolve, formed a quantitative, nuanced picture of how the burgeoning field of gene therapy is understood and interpreted by key stakeholders, providing recommendations to improve communication and enhance awareness as this important technology transitions into the mainstream of rare disease treatment.

Physician and Patient Perceptions Regarding Physician Training in Rare Diseases: The Need for Stronger Educational Initiatives for Physicians (Engel P., Bagal S., Broback M., Boice N.; 2013)