Florida State University Pediatric Rare Diseases

FSU Institute for Pediatric Rare Diseases

Florida State University has recently launched the Institute for Pediatric Rare Diseases, an establishment dedicated to advancing research and developing treatments for unusual childhood diseases. The institute, which will fill a crucial gap in the healthcare industry, was made possible thanks to the actions of Rep. Adam Anderson, who pushed for $1 million in funding following the tragic loss of his son Andrew, who sadly died from Tay-Sachs Disease; a rare disease that affects less than 100 people annually in the United States.

Anderson, a Florida State alumnus, explained with gratitude:

“Since our son Andrew’s diagnosis of Tay-Sachs Disease in 2016, my wife Brianne and I have made it our mission to do everything in our power to ensure other families don’t have to live that same nightmare that we did, but sadly many families do...
There are currently 7,000 known rare diseases that impact over 350 million people globally and 30 million people nationwide. To put that into perspective, that’s nearly 10% of the population of our country and more than the entire population of Florida. So, while we call them rare, when looked at as a whole they are more frighteningly common than you think.”

Adam Anderson

Adam Anderson is a conservative local businessman, husband & father, Representing N. Pinellas Dist 57. Rep. Adam Anderson ran for public office to fund research into rare diseases like the one that affected his son

The launch of the Institute for Pediatric Rare Diseases center at the College of Medicine, which was also supported by Gov. Ron DeSantis, was recently celebrated at the Florida State University Day held at the state Capitol 1st Feb 2024.


More Research and Better Treatment for Pediatric Rare Diseases

The institute brings together scientists, clinicians, and educators to leverage the latest in immune response research and the rapidly advancing field of gene therapy. Its goal is to tackle the common research challenges that face pediatric rare diseases.

With significant advancements in recent gene therapy and considerable breakthroughs in gene editing techniques such as CRISPR, the institute is confident its efforts will build upon the current knowledge base to play a critical role in the future treatment of the more than 15 million children in the USA living with such conditions.

Led by Director Pradeep Bhide and the Jim and Betty Ann Rodgers Eminent Scholar Chair of Developmental Neuroscience, the institute will be located at FSU College of Medicine. The team will focus on discovering the root causes of many different pediatric rare diseases so that researchers can take these findings and develop effective new diagnostic and treatment tools. The institute also plans to create a DNA sequencing facility and launch a graduate program for those with experience in genetic counseling. Bhide explained:

“This is a unique opportunity for our faculty and students to join forces and work together to develop novel technologies for the diagnosis and treatment of pediatric rare diseases...

We deeply appreciate the support of the State of Florida in funding this research for the benefit of all children and families — not just here in Florida, but everywhere. We have made tremendous progress in just a short time, and Florida can truly make a difference in the lives of our youngest citizens. We are grateful to Gov. DeSantis, Rep. Anderson and the entire Florida Legislature for their support of our work.”

There are currently 7,000 rare diseases that affect over 350 million people worldwide. These low numbers offer little incentive for scientists and pharmaceutical companies alike to invest their time and efforts into the research required to develop effective treatments, which, in a twisted turn of fate, is also more challenging since there’s little knowledge base to build upon.

But in this specific case, the first-of-its-kind center is personal to the Palm Harbor Republican and, without his efforts, would likely not have been created. So it’s with many thanks that we bless the launch of the Institute for Pediatric Rare Diseases and wish all the best for its future discoveries and developments.

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