Robert J. Gorlin Symposium

Precision Metrics for Cognition

Program Chair: Elsa Shapiro, PhD
February 6, 2022
3:00 – 5:00 PM Pacific Standard Time (PST)

Robert J. GorlinThe Robert J. Gorlin Symposium, will honor the work of Robert James Gorlin, DDS, PhD. Dr. Gorlin was an maxillofacial pathologist, geneticist and academician at the University of Minnesota School of Dentistry. His groundbreaking research in genetic disorders of the head and neck, spanning over 50 years, revolutionized the understanding of the morphology of lysosomal diseases and many other genetic disorders. The inaugural Robert J. Gorlin Symposium, Precision Metrics for Cognition, will focus on groundbreaking metrics to measure cognitive function in children with lysosomal diseases.

About the Session

This new session at WORLDSymposium 2022 is intended to discuss the urgent need to develop and implement precise metrics for measuring cognitive change in children with lysosomal diseases. Precision Metrics for Cognition will bring together leading experts to provide an explanation and rationale for using growth scale values (GSVs) to measure developmental longitudinal cognitive change in children with early onset and severe lysosomal diseases.

Through examples and case studies, experts will share more about the need for increased precision in measuring cognitive change and how GSVs are useful when facing the challenge of revealing the developmental trajectory of young children.

The goal of this session is to provide attendees with insight into the perspective of regulatory bodies, and determine how to apply this approach in their research to reliably and precisely measure cognitive change.

$50 Registration Required.

Session Sponsors

This session is Sponsored by:

“JCR
“Orchard
“Takeda

Learning Objectives

  • Review the range of metrics for measuring cognitive and adaptive outcomes.
  • Discuss Growth Scale Values (GSVs) and their historical development.
  • Differentiate the advantages and limitations of GSVs.
  • Analyze how GSVs and other metrics have been used in clinical trials and natural history studies and what additional data is needed to validate these metrics.
  • Summarize the regulatory perspective on expectations for metrics in clinical trials.

Preliminary Agenda

3:00 PM
Introduction and insights
Elsa Shapiro, PhD, ABPP

3:15 PM
Growth scale values (GSVs): theory, development, and characteristics
Mark H. Daniel, PhD

3:30 PM
Advantages of GSVs in clinical trials
Adam Scheller, PhD

3:40 PM
Measuring cognitive outcomes with GSV’s in MPS I
Julie B. Eisengart, PhD, LP

3:50 PM
Use of GSV scores in natural history of MPS IIIB
Bernice Kuca, MS

4:00 PM
Use of GSVs using the Vineland in CLN3 Batten disease
Heather Adams, PhD

4:10 PM
Measuring cognitive and developmental outcomes in trials for neuronopathic lysosomal diseases
Patroula Smpokou, MD

4:25 PM
Panel Discussion and Audience Q&A

5:00 PM
Adjourn

Faculty

Elsa Shapiro, PhD, ABPP (CHAIR)
Shapiro Neuropsychology Consulting, LLC
Professor of Pediatrics and Neurology
University of Minnesota
Portland, OR

Mark H. Daniel, PhD
Consultant
Mark Daniel Services, LLC
Blaine, MN

Adam Scheller, PhD
Licensed Psychologist
Director, Pharma Research Services
Pearson Clinical Assessment
Orlando, FL

Julie B. Eisengart, PhD, LP
Associate Professor Department of Pediatrics
Director Neurodevelopmental Program in Rare Disease
Division of Clinical Behavioral Neuroscience
University of Minnesota
Minneapolis, MN

Bernice Kuca, MS
Head of Clinical/Regulatory Operations
Allievex Corporation
Boston, MA

Heather Adams, PhD
Associate Professor
Department of Neurology – Division of Child Neurology
University of Rochester Medical Center
Rochester, NY

Patroula Smpokou, MD  
Deputy Director
Division of Rare Diseases & Medical Genetics (DRDMG)
Office of New Drugs, CDER, FDA
Washington, DC