Despite significant progress in enzyme replacement therapies and novel gene therapies, it remains true that for many of the more than 7,000 rare diseases impacting more than 300 million people worldwide there are no effective therapies.
For many of these diseases diagnosis is difficult, complex and ineffective. However, with increased biotech advances, investment at an all-time high, improved interest from Pharma, and a stronger than ever voice from patient advocates, there has never been a more significant time to join in the fight against rare disease.
As proud corporate supporters of both NORD and Eurordis, we are committed to working together with key stakeholders to do all we can to accelerate clinical research for rare diseases. On Rare Disease Day, Syneos Health will be hosting a Rare Disease Day Symposium Thursday, Feb. 28th from 11:30-3:00 PM at our new global headquarters in Morrisville, NC.
The Symposium will bring together some of the thought leaders in the rare disease space for thought-provoking presentations and candid discussion about the present and future state of clinical research in rare disease. We will feature a “360” perspective of voices from patients, legislators, regulatory experts, physicians and sponsors as well as the latest local and national initiatives driving change.
Representatives from the NC State Legislature, NC Rare Disease Advisory Council, UNC Medicine, NORD, patient advocacy, as well as biotech and policy leaders will lead an interactive panel discussion to develop actionable ways in which we can better collaborate to support patient needs.