Rare Disease Partnering Summit & Reception

Don’t miss the opportunity to hear these exciting Business Forum presentations by pediatric institutions.

  • Rare Disease Partnering Summit: Presentations 2:00 – 5:00 pm
  • Rare Disease Partnering Summit Reception, sponsored by Shire: 5:00 – 6:00 pm

Join renowned investigators from five world-class pediatric institutions on April 24 from 2-5pm as they present translational research projects and partnering opportunities that focus on rare diseases. The Rare Disease Partnering Summit is designed to facilitate collaboration with industry and advance research in rare and orphan diseases. A networking reception will follow at 5pm. Participating institutions include Boston Children’s Hospital, Cincinnati Children’s Hospital, Children’s Hospital Los Angeles, The Hospital for Sick Children, Toronto and University of Chicago Comer Children’s Hospital.

Open to all attendees.

Rare Disease Partnering Summit Presentations 2:00 – 5:00 pm


2:00 pm
Introduction from Shire


2:05 – 2:35 pm David Margulies
Boston Children’s
David Margulies, MD
Title: Towards Precision Care



Clifford Woolf, MD, PhD 

Title: Stem Cell Derived Human Neurons For Modeling Disease and Drug Screening


2:40 – 3:10 pm
Cincinnati Children’s
Peter Margolis, MD, PhD 
Title: The Collaborative Chronic Care Network: A Technology-Enabled Learning Health System for Improvement, Innovation and Research




Bruce Trapnell, MD
Title: TBD






3:15 – 3:45 pm
Children’s Hospital Los Angeles
Timothy Triche, MD, PhD
Title: Curing Childhood Cancer: A Better Way?




Barry Maurer, MD, PhD
Title: Patented Nanotechnology Fenretinide Formulations Drive Novel Ceramide-Based Therapy and Sustained Complete Responses




3:50 – 4:30 pm Physician Photo
University of Chicago Comer Children’s
Erika Claud, MD
Title: TBD




Beth McNally, MD, PhD Physician Photo
Title: TBD





4:25 – 4:55 pm  Dr. Peter Dirks
Toronto SickKids
Peter Dirks, MD, PhD
Title: Dissecting Brain Tumor Heterogeneity: Implications for Novel Novel Therapies





Don Mahuran, PhD Don Mahuran
Title: Small Molecule Therapy for Lysosomal Storage Diseases






For more infotmation visit http://convention.bio.org/rare-diseases-partnering-summit/