Presentation of the Annual Award for Leadership in Personalized Medicine

Emily Kramer-Golinkoff, a cystic fibrosis patient and co-founder of Emily’s Entourage, is the winner of this year’s Award for Leadership in Personalized Medicine.

More than 2,000 mutations in the CFTR gene can cause cystic fibrosis, a progressive disease that results in severe damage to the lungs, digestive system, and other organs. Available drugs can improve how the CFTR protein functions in 90 percent of cystic fibrosis patients. But for Emily and thousands of other cystic fibrosis patients who suffer from the rarest forms of this devastating disease, life is a perpetual struggle against an unjust genetic enemy that always has the upper hand.

Despite the interruptions of breathing treatments, injections for cystic fibrosis-related diabetes, and a medication regimen that requires 30 pills a day to delay end-stage disease for as long as possible, Emily has played a leading role in engineering a new future for patients, like her, who do not benefit from any of the existing disease modulating treatments.

After earning bachelor’s and master’s degrees in communications and bioethics from the University of Pennsylvania, Emily co-founded Emily’s Entourage in 2011. The nonprofit institution has since awarded more than $9.8 million in funding for research and drug development programs designed to develop life-saving treatments and cures for the last 10 percent of cystic fibrosis patients. Through its grant programs, philanthropic investments, clinical trial recruitment activities, and patient registry, the organization has brought potentially life-altering experimental therapies to hundreds of patients who would otherwise have few meaningful treatment options.

“Personalized medicine is my hope, and it is the hope of every single person like me with a rare disease or a rare mutation or a medical condition that simply requires a more customized treatment,” Emily says of the cause.