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)      Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN): Metachromatic Leukodystrophy

• Laura Adang, MD, PhD, MSTR, Assistant Professor of Child Neurology, Children’s Hospital of Philadelphia, Philadelphia, PA, USA

  • Presentation slides

3)      International Niemann-Pick Disease Registry (INPDR), Sharing Data to Advance Research in Niemann-Pick Diseases 

• Shaun Bolton, Chief Operating Officer, INPDR, United Kingdom

  • Presentation slides

4)      Data Without Borders:  Proactive Data Sharing in a Not-for-Profit Clinical Research Organization

• Ozlem Goker-Alpan, M.D, Founder and CMO, Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), Fairfax, VA, USA

  • Presentation slides

5)      Questions & Discussion

• Presenters panel: Laura Adang, MD, PhD, MSTR, Shaun Bolton, Ozlem Goker-Alpan, M.D, 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