Presentations

Click on the links below to view the slides presented by several of our speakers.

Speaker Presentation Title
Heather R. Adams Outcome Measures and Infrastructure for Phase III Studies in Batten Disease (JNCL)
Jonathan W. Mink Natural History of JNCL and other NCLs
Robert C. Griggs The Experimental Therapeutics of Rare Diseases
Heather R. Adams Development and Validation of Clinical Trial Endpoints
Lisa de Blieck Patient Registries in Rare Diseases
Erika F. Augustine Widely Used Clinical Endpoints for Neurologic Disease – Applications for JNCL
Ellen Bletsoe Phase III Clinical Trials in JNCL: Treatment Outcomes & What is Most Meaningful to Families
Lori Sikorra Take Your Time
Michael P. McDermott Clinical Trials in Rare Diseases: Challenges in Design, Analysis, and Interpretation
David Pearce Preclinical Endpoints in CLN3
John F. Staropoli The MGH NCL Biorepository: Development of Preclinical Screening Platforms and Biomarkers
Angela Schulz DEM-CHILD (for information about DEM-CHILD, please see the website for the project)
Angela Schulz Review of Hamburg NCL Rating Scale
Jonathan W. Mink Review of UBDRS in JNCL: Reliability, Validity, and Endpoints

Support for this conference was provided by:

The National Institutes of Health – National Institute of Neurological Diseases and Stroke (NIH/NINDS): 1R13 NS078981, Outcomes and Infrastructure for Phase III Clinical Trials in JNCL - Batten Disease

The University of Rochester Child Neurology Research Fund