Agenda:

1)      Welcome, Goals for the Workshop Series, Overview

• Krista Casazza PhD, RD, CSSD, C-Path & Cara M. Weismann, PhD, Orphan Disease Center

  • Presentation slides

2)      Preparing for Retroactive Data Sharing in Batten’s Disease, BDSRA Foundation

• Noah Siedman, Community & Database Manager, BDSRA Foundation, Gahanna, OH, USA

  • Presentation slides

3)      Working with Sponsors to Encourage Retroactive Data Sharing in Lysosomal Storage Disorders, CPLD

• Krista Casazza PhD, RD, CSSD, Scientific Director, Pediatrics, CPLD, C-Path, Tucson, AZ, USA

  • Presentation slides

4)      Rare Disease Cures Accelerator- Data Analytics Platform: Role in Accelerating Data Sharing and Rare Diseases Drug Development.

• Heidi Grabenstatter, PhD, Scientific Director, Rare Disease Cures Accelerator – Data Analytics Platform (RDCA-DAP), Tucson, AZ, USA

  • Presentation slides

5)      Natural History Study of the Gangliosidoses

• Jeanine Jarnes, PharmD, MSc, BCOP BCPS, University of Minnesota, Department of Pediatrics, Minneapolis, MN, USA

  • Presentation slides

5)      Questions & Discussion

• Presenters panel: Noah Siedman, Heidi Grabenstatter, PhD, Jeanine Jarnes, MD, PhD, Cara M. Weismann, PhD, & Krista Casazza PhD, RD, CSSD

6)      Summary, Wrap up, Future Directions

• Cara M. Weismann, PhD, Orphan Disease Center & Krista Casazza PhD, RD, CSSD, C-Path