Fueling Advancements in Rare Disease: The Power of Partnerships and the Patient Voice

At the end of the evening, Ja’cori (l) brought the house down with his song that reflects the love, worry and sunny outlook of a little boy’s life with CGD.

Last week, to honor Rare Disease Day, thousands of people around the globe took time to advocate and raise awareness about rare diseases and their impact on patients’ lives. With only five percent of all rare diseases having an FDA approved treatment, there is tremendous unmet need that requires innovative collaborations and continued conversations across the healthcare spectrum. As part of the day, a robust forum was held in Washington DC to discuss the challenges and opportunities in rare disease research. Among the people attending was a spirited 10-year-old boy, Ja’cori, who lives with chronic granulomatous disease (CGD). Ja’cori’s journey with CGD, which was transformed into song that reflects the love, worry and sunny outlook of this little boy’s life, closed the evening and brought the house down. To learn more about the inspiring evening,  click here. To listen to Ja’cori’s song, click here.

Posted in:

Leave a Reply