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Center for Rare Disease Seminar Series: Scaling Precision Therapeutic Development for Rare Disease

Location: Hybrid: Health Learning Building Auditorium (HLB 1.111) & Zoom

Address: 1501 Red River St., Austin, Texas 78712 | View Map »

Date: Thursday, April 17, 2025

Time: 12–1 p.m.

Contact: Suzette Nava

Category:

About the Event

In this installment of the Center for Rare Disease Seminar Series co-hosted by the Center for Rare Disease and the Department of Pediatrics, Marni J. Falk, M.D., presents “Scaling Precision Therapeutic Development for Rare Disease: Implementing Effective Pathways To Bridge From Preclinical Modeling to Clinical Trials.”

The talk provides scientists and clinicians with a roadmap to advance precision therapies in rare diseases by validating predictive preclinical models, integrating biomarker development and designing efficient first-in-human trials for rapid bench-to-bedside progression.

Falk is an attending physician and executive director for the Mitochondrial Medicine Frontier Program at Children’s Hospital of Philadelphia. She also holds the distinguished endowed chair in the Department of Pediatrics and is a professor in the Division of Human Genetics within the Department of Pediatrics at the University of Pennsylvania Perelman School of Medicine.

For more information and the Zoom link, contact Suzette Nava.