Celebrating Rare Disease Day

Tune in to hear Emily Schaller – patient advocate, PPD Beach2Battleship Hero and a founder of the Rock CF Foundation – share her perspective on life-changing clinical research.  

In 1983, a baby born with cystic fibrosis wasn't expected to live long enough to graduate from high school. Thirty years ago, this was the conversation doctors were forced to have with Emily Schaller's parents.

Knowing their daughter had an uncertain future, but unwilling to give up hope, Emily's parents proceeded with the standard care of the day: antibiotics and chest physical therapy, the repeated pounding of the chest and back to break up accumulating mucus in the lungs.

When Emily was a teenager, her parents enrolled her in her first clinical trial, and over the years, she has participated in many. Recently, Emily finished a trial for an oral drug that was approved by the U.S. Food and Drug Administration. Within four days of starting the Phase III trial, she was able to take a full deep breath – without coughing. Her lung function soared to record numbers. Her junky cough nearly disappeared.

Thanks to advancements in clinical research for rare diseases, Emily is now part of a growing population of adults living and thriving with cystic fibrosis. Once it was considered a childhood disease because CF patients rarely made it to adulthood. So it was with no small measure of gratitude that Emily recently joked with PPD employees in Wilmington that she had started researching retirement accounts! Just days before celebrating her 33rd birthday, Emily spent a day at PPD headquarters sharing her patient experience and encouraging employees to keep up their hard work in advancing drug development. She's alive today because of it!

On Saturday, 28 February 2015, people around the world will celebrate the eighth international Rare Disease Day®. The goal of Rare Disease Day is to bring people living with rare diseases out of the shadows and into the light. Emily's story is just one of many from individuals who suffer from life-threatening rare diseases with no known cure and little awareness. Clinical research is instrumental in finding cures for these illnesses. Consider a career with PPD advancing drug development and be part of the solution!