Kakkis Tandon
Kakkis Tandon
Multi-Domain
Responder Index
Multi-Domain
Responder Index
Clinical Trial Model
Clinical Trial Model
Embedded Files
Responder Index
A New Way to Evaluate Drugs for
Rare & Ultra Rare Diseases (and beyond)
Scientific Leaders
Scientific Leaders
CEO and President, Ultragenyx
SVP, Biometrics &
Development Strategy, Ultragenyx
MDRI Overview
MDRI Overview
Presentations
Presentations
Translating Rare Disease Research into Medicine
The Orphan Drug Act Myth: The Role of Prices Versus ODA Incentives
The Challenges of Managing Heterogeneity in Rare Diseases
Scientific Method
Scientific Method
Emil D. Kakkis, M.D., Ph.D.
Scientific Publication
Scientific Publication
The Multi-Domain Responder Index: A Novel Analysis Tool to Capture a Broader Assessment of Clinical Benefit in Heterogeneous Complex Rare Diseases
Emil D. Kakkis, M.D., Ph.D. and P. K. Tandon, Ph.D.
2021
New Data Report
New Data Report
Report by Pomona College Statistics student, Uras Uyal (Ultyragenyx Research Intern Summer 2025) and Prof. Jo Hardin, PhD
June 30, 2025
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