Emily has worked with the Foundation as a volunteer on a number of different initiatives and is such an inspiration within the cystic fibrosis community. 

An important honor, Champions of Change are ordinary Americans doing extraordinary things in their communities. Along with eight other honorees, Emily participated in a day of panels, meetings and speeches all focused on the idea that the delivery of medicine can be targeted to a specific person's genetics, lifestyle and environment. 

We were invited to attend this exciting event at the historic Eisenhower Executive Office Building.

The main event began with opening remarks by Senior Advisor to the President, Brian Deese. We were also fortunate to hear remarks by Dr. Francis Collins, director of the National Institutes of Health (and member of the team that discovered the cystic fibrosis gene in 1989), among others. 

In a panel on precision medicine, Emily beautifully articulated the realities of living with cystic fibrosis. As someone with two copies of a rare nonsense mutation, she talked about working to advance research into this rare form of cystic fibrosis and the power that patients can wield in the search for treatments and cures.

The event is part of President Obama's Precision Medicine Initiative (PMI). Precision medicine is an emerging approach to disease treatment and prevention that takes into account each person's genetic makeup and other factors. 

New targeted therapies like Kalydeco and Orkambi that treat CF patients with specific mutations in the CFTR gene are wonderful examples of precision medicine. 

Indeed, when the President announced the initiative at the 2015 State of the Union, he noted that, “In some patients with cystic fibrosis, this approach has reversed a disease once thought unstoppable.”  The White House has further highlighted how taking the precision medicine drug Kalydeco has markedly improved the health of CF patient Bill Elder, Jr. Bill had the honor of sitting with first lady Michelle Obama at the State of the Union address.

This is a time of great hope and optimism in the cystic fibrosis community, as we discover new ways to treat this disease and embark on the path toward a cure. Emily's story represents the innovative thinking that is going to get us where we need to go.

Check out Emily's post, “Emily's Entourage: Giving Hope to Orphans of Orphan Disease,” on the White House's blog.