IPRD & A.J.Anderson
Foundation Launch Powerful
Rare Disease Event
2026 Rare Disease Symposium
The Florida Institute for Pediatric Rare Diseases (IPRD) partnered with the A.J. Anderson Foundation to host a two‑day inaugural event at the Innisbrook Resort in Palm Harbor on June 3rd – 4th, 2026.
Commitment to elevating
The program featured a Rare Disease Symposium, The A.J. Anderson Dinner for a Cause, and The A.J. Anderson Golf Tournament. This inaugural event marks the beginning of a long‑term partnership between IPRD and the A.J. Anderson Foundation, united in their commitment to elevating rare‑disease voices and accelerating meaningful progress for affected children and their families.
Rare Disease Symposium
More than 200 participants, including families, researchers, clinicians, and advocates, came together to share knowledge, build community, and advance the mission of improving outcomes for children affected by rare diseases. On June 3, 2026, the A.J. Anderson Foundation and Florida IPRD cohosted a rare disease conference, offering updates on current research, clinical progress, and the future of treatment. View the Symposium Program Booklet.
Keynote Speaker David Bick, M.D.
“Screening 100,000 Newborns Using Whole genome Sequencing: Early Results from Genomics England’s Generation Study”
David Bick, M.D., is an internationally recognized leader in clinical genomics and genomic medicine, with experience in pediatrics, clinical genetics, and molecular genetics. He serves as Principal Clinician for the Newborn Genomes Programme (Generation Study) at Genomics England, where he is helping evaluate the integration of whole genome sequencing into newborn screening across the United Kingdom’s National Health Service.
Session 1:
Genomic Newborn Screening
Moderated by IPRD Senior Associate Director for Precision Medicine David Ledbetter, Ph.D., FACMG
Leaders in genomic medicine shared forward‑looking insights across four key sessions. In “The Cost of Underdiagnosis,” Britt Johnson, Ph.D., FACMG, Senior Vice President of Medical Affairs at GeneDx, highlighted the clinical and financial burden created by delayed or missed rare‑disease diagnoses and emphasized the value of early genomic testing.
Session 2:
Advances in Gene Therapy
Moderated by IPRD Director Pradeep Bhide, Ph.D.
Session 2 spotlighted emerging pathways and future directions in gene therapy for rare and ultra‑rare diseases. Miguel Sena-Esteves, Ph.D., Associate Professor in the Department of Genetic and Cellular Medicine, UMass Chan Medical School, discussed “An Alternative Pathway for Academia-Led Development of AAV Gene Therapies for Ultra-Rare Diseases”, outlining how academic centers can drive innovation for conditions.
Dinner for a Cause
Following the symposium, two additional events took place; the A.J. Anderson Dinner for a Cause and the A.J. Anderson Golf Tournament, both of which were incredible successes.
During the dinner, Rep. Adam Anderson and his family presented awards honoring individuals and families affected by rare diseases. The evening highlighted the strength, resilience, and unity of the rare disease community, bringing together families, advocates, and supporters in a meaningful and uplifting way.
Thank you to our Sponsors!
