When it comes to rare disease, rare isn’t actually that rare. More than 10,000 rare diseases have been identified. Rare diseases affect more than 350 million people across the globe and 30 million people in the United States alone. Of those in the United States, approximately 50% are children. As more genetic conditions are discovered, this number is only expected to rise.
Florida State University’s College of Medicine is working to transform the landscape for those affected by rare diseases. According to Kathleen Haughney in Florida State University News, the University recently celebrated the launch of the Institute for Pediatric Rare Diseases.
The Institute for Pediatric Rare Diseases was funded by $1,000,000; the push for funding, eventually granted by the Florida State Legislature, was spearheaded by Rep. Adam Anderson (R-Palm Harbor), who lost his son Andrew to a rare inherited disorder called Tay-Sachs disease.
The Institute for Pediatric Rare Diseases: How It Will Operate
Florida State University’s College of Medicine describes its mission as a push to:
educate and develop exemplary physicians who practice patient-centered health care, discover and advance knowledge, and are responsive to community needs, especially through service to elder, rural, minority, and underserved populations. The FSU College of Medicine will lead the nation in preparing compassionate physicians to deliver the highest quality 21st Century patient-centered medicine to communities of greatest need.
As such, faculty from the College of Medicine see the Institute for Pediatric Rare Diseases as a way to reach out and respond to the needs of rare disease families in Florida.
The Institute for Pediatric Rare Diseases will look to advance research into rare pediatric diseases. In the United States, rare conditions are those affecting fewer than 200,000 people. Through this research, the institute aims to encourage scientists and researchers to collaborate and facilitate increased understanding into these conditions. Further, through interdisciplinary exploration and hands-on evaluation, it also aims to identify and potentially develop therapies to meet the needs of children within the rare disease community.
Eventually, the institute plans to launch further programs and research into genetics, gene therapies and genetic counseling, and DNA sequencing. Ultimately, this institute is poised to cultivate a stronger medical understanding of these conditions while improving quality-of-life on a broader scale.
