Boston Children's Hospital 2nd Annual Rare Disease Symposium

Friday, October 21, 2011
Folkman Auditorium, Enders Building (320 Longwood Ave., Boston, MA)
9 am - 5 pm
Breakfast served

If you are a non Children's employee and would like to attend the Symposium, please RSVP to TIDO@childrens.harvard.edu or call (617) 919-3022.

Download the flyer (PDF)

Co-sponsored by the Technology and Innovation Development Office and the Manton Center for Orphan Disease Research (the only research center in the world focused on the study of rare disorders), this event is organized to bring the Children’s rare disease community together with external organizations, particularly in industry and government, that support development of treatments for rare diseases.

The event will highlight laboratory and clinical research projects that address rare diseases at Children’s, and will provide information about best practices and resources available to support your work furthering research and therapies that address rare disease.

Industry interest in rare diseases has grown dramatically in the recent past, and this year’s event is eagerly anticipated by our industry colleagues. Senior executives from Pfizer, Sanofi (Genzyme), J&J, Shire, Novartis and numerous venture capital firms have confirmed that they will attend and look forward to meeting with you.

If you can't attend the symposium in person, watch online here (sign in as guest).

8:30 AM - Sign in and breakfast

9 AM - Boston Children's Presentations:

10:40 AM - Coffee break

10:55 AM - Boston Children's Presentations:

Noon - Lunch & Poster Session, Inn at Longwood Medical, Fenway Room

1:15 PM - Afternoon Boston Children's Panel:

Translational Medicine in the Academic Setting

Hear from three Children’s investigators who each have a unique story of bringing a scientific discovery to the clinic here at Children’s. How did they do it? What support did they receive along the way? The panelists will share hurdles, best practices, tips for other investigators who want to move their research to clinic, and thoughts on external partnerships that can speed development of therapies for patients.


2:30 PM - Coffee break

2:45 PM - Afternoon External Panel:

Partnerships to Support Rare Disease Therapeutic Development

Why is industry increasingly interested in rare disease therapeutics and how can external organizations help Children’s investigators develop these therapies? Expert panelists representing both industry and government will discuss the underlying dynamics of this commercial interest and the incentives, the unique partnership programs and infrastructure support that each can provide to academic investigators.


  • Randall Carpenter, MD - CEO; Seaside Therapeutics
  • Gerald F. Cox, MD, PhD - Vice President of Clinical Development; Genzyme, a Sanofi Company
  • Nora Yang, PhD, MBA - Director of Strategic Planning and Program Operations; NIH Center for Translational Therapeutics for Rare and Neglected Diseases
  • Christine Mueller, DO - Medical Officer and Outreach Coordinator; FDA’s Office of Orphan Products Development
  • Moderator: Eric Nelson, PhD - Partner; NCE Biotech, LLC

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