Speaker Profile
M.D., Ph.D, Professor, Department of Pediatrics, Neurological Sciences, Biochemistry, Rush U. Medical Ctr.
Biography
Elizabeth Berry-Kravis established the Fragile X Clinic and Research Program at Rush University Medical Center in 1992 and has provided care to over 800 patients with fragile X syndrome (FXS). She conducts research on FXS, including genotypephenotype, molecular, biomarker, outcome measure, and natural history studies, and clinical trials. She has expanded this clinical and translational work to other neurogenetic diseases in the past 10 years, including work on PMS, NPC, Angelman syndrome, Rett syndrome, Battens disease, PKAN, and creatine transport deficiency. She has led the effort to develop new targeted treatments for FXS and other genetic neurological diseases, and has implemented novel trial designs, including Nof1 trials.
Session Abstract – PMWC 2024 Silicon Valley
Track Co-Chairs:
Yael Weiss, Mahzi Tx
Peter Marks, FDA
- PMWC 2024 CGT Award Ceremony
Pioneer Honoree: Katherine A. High, AskBio
Pioneer Honoree: Timothy Yu, Boston Children's Hospital
- Piloting Interventional Genomics for Orphan Diseases
Keynote: Timothy Yu, Boston Children's Hospital
- Accelerated Approval for Small Populations: Biomarkers & Endpoints (PANEL)
Chair: Peter Marks, FDA
- Jennifer Puck, UCSF
- Katherine A. High, Rockefeller University
- Timothy Yu, Boston Children's Hospital - Novel Clinical Design Approaches in Rare Diseases (PANEL)
Chair: Adam Shaywitz, Bridge Bio
- Salvador Rico, Encoded Therapeutics
- Eric Crombez, Ultragenyx
- Elizabeth Berri Kravis, Rush University
- Empowering Patient Advocacy in Rare Disease Therapies (PANEL)
Chair: Yael Weiss, Mahzi Tx
- Charlene Son Rigby, STXBP1 Foundation
- Michael Graglia, Syngap Research Fund - Advancing the Frontier: Gene and Cell Therapies for Rare Diseases (PANEL)
Chair: Matthew Porteus, Stanford
- Swati Tole, Capsida Biotherapeutics
- Adrian Veres, Dyno Therapeutics
- Thomas Wechsler, J&J
- David Lebwohl, Intellia Therapeutics
- Pooja Agarwal, BioMarin Pharmaceutical
