"A significant part of our work is building partnerships with families who are affected by a rare disease and supporting them throughout the clinical trial process. Many rare diseases are genetic, with greater than 50% affecting children. This reality demands a new model in which partnerships with advocacy organizations to embed patient perspectives become a critical means for us to understand the patient journey and better inform and engage patients, their families, and caregivers throughout the trial. This deep collaboration with our patient populations and their advocacy groups enables us to advance closer to the goal: to develop treatments that improve longevity and quality of life for patients with rare diseases.”
Infectious Diseases