ODC - Hosted Symposia
• ZC4H2 Think Tank, September 2018
o Dubai, UAE
o 14 attendees
o In partnership with The ZC4H2 Deficiency Research Foundation
• International Crigler-Najjar Syndrome Symposium, June 2018
o London, UK
o 30 attendees
• Alnylam Rare Disease Workshop “Exploring Liver Applications for RNAi Therapeutics”
o In partnership with Alnylam
• Genome Editing for PCSK9, February 2018
o Philadelphia, PA
o 9 attendees
• SMA Patient Advisory Board Meeting – Gene Therapy for Spinal Muscular Atrophy, January 2018
o Philadelphia, PA
o 10 attendees
• CDKL5 Forum, November 2017
o Hosted by the Loulou Foundation, in partnership with ODC
• ALS Advisory Think Tank, September 2017
o Boston, MA
o 11 attendees
• MPS Symposium, July 2017
o Philadelphia, PA
o 60 attendees
o Sponsored by REGENXBIO and Lysogene
• RARE Patient Advocacy Symposium, May 2017
• UPenn Hearing Loss Symposium, April 2017
o Philadelphia, PA
o 33 attendees
• Pig Models of Human Orphan Diseases, December 2016
o Philadelphia, PA
o 50 attendees
• Spinal Muscular Atrophy (SMA) Symposium on Optimizing Care, September/October 2016
o Philadelphia, PA
o 71 attendees
o Sponsored by AveXis, Inc.
• UPenn Crigler-Najjar Syndrome Symposium, June 2016
o Philadelphia, PA
o 45 attendees
• RARE Patient Advocacy Symposium, May 2016
• Castleman Disease Symposium, November 2015
• UPenn DMD Gene Therapy Symposium, April 2015
o Philadelphia, PA
o 55 attendees
o In partnership with Parent Project Muscular Dystrophy
• Penn LAM Symposum - Lymphatics, Lymphangiogenesis, and VEGF-D: Targets for LAM, February 2015
o Philadelphia, PA
o 23 attendees
o In partnership with The LAM Foundation
• Drug Discovery for Nonsense Mutations in Cystic Fibrosis, January 2015
o Philadelphia, PA
o 30 attendees
o In partnership with Emily’s Entourage
• LSD Symposium, September 2010
o Philadelphia, PA
o 90 attendees
ODC - Sponsored Symposia
• Therapeutic Development for Nonsense Mutations: The Final Frontier of CF, September 2018
o Philadelphia, PA
• Deciphering Beckwith-Wiedemann Syndrome, July 2017
o Philadelphia, PA
o In partnership with Children’s Hospital of Philadelphia and Alex’s Lemonade Stand Foundation
• Angelman Syndrome Gene Therapy Think Tank, December 2016
• International Summit on idiopathic multicentric Castleman Disease (iMCD), November 2016
o Philadelphia, PA
o In partnership with the Castleman Disease Collaborative Network
• Congenital Hypoglycemia Disorders: Hyperinsulinism and GSD, April 2016
o Philadelphia, PA
o In partnership with The Congenital Hyperinsulinism Center Division of Endocrinology and Diabetes
