Experts From Government, Industry, Patient Advocacy to Speak at U.S. Conference on Rare Diseases and Orphan Products

Sep 28, 2012, 12:22 ET from National Organization for Rare Disorders (NORD)

"Shaping the Future Now" is Theme; Speakers will Address Current Political and Economic Climate

WASHINGTON, Sept. 28, 2012 /PRNewswire-USNewswire/ -- Patient advocates, industry leaders, medical researchers, government professionals and investors will address together "Shaping the Future Now" at the 2nd annual U.S. Conference on Rare Diseases and Orphan Products in Washington, DC October 22 – 24.


Sponsored by the National Organization for Rare Disorders (NORD) and Drug Information Association (DIA), this major event for all stakeholders in the field is presented in collaboration with the National Institutes of Health (NIH), Food and Drug Administration (FDA), Rare Diseases Europe (EURORDIS) and Duke School of Medicine Department of Pediatrics.

"This conference is for everyone with an interest in rare diseases and orphan products," said Peter L. Saltonstall, President and CEO of NORD.  "It is unique in that it draws together all stakeholders to address the shared challenge - how to accelerate the development of safe, effective treatments for people with rare diseases."

The three-day event to take place at Washington's Capital Hilton Hotel will feature plenary sessions as well as smaller group workshops organized according to three themes: Policy, Research and Regulation, and Special Challenges in Rare Diseases.

There will be sessions led by FDA senior staff for researchers involved in orphan product development, workshops for patient advocates, and presentations on the current economic climate for orphan product investment.

Online registration is now open.  Early bird rates are in effect until October 1.  Speakers this year will include:

  • Stephen P. Spielberg, MD, PhD, FDA Deputy Commissioner
  • Christopher P. Austin, MD, Director, National Center for Translational Sciences
  • Preston W. Campbell III, MD, Executive Vice President for Medical Affairs, Cystic Fibrosis Foundation
  • Mark McClellan, MD, PhD, Director, Engelberg Center for Health Care Reform, The Brookings Institution
  • Susan Riley Keyes, PhD, JD, Vice President, Nemucore Medical Innovations, Inc.
  • Robert M. Califf, MD, Vice Chancellor for Clinical and Translational Medicine and Director, Duke Translational Medicine Institute
  • Anne R. Pariser, MD, Associate Director for Rare Diseases, Office of New Drugs, CDER, FDA
  • Charles A. Mohan, CEO/Executive Director, United Mitochondrial Disease Foundation
  • Jonathan S. Leff, Managing Director, Warburg Pincus
  • Jayne Gershkowitz, Senior Director, Patient Advocacy & Public Policy, Amicus Therapeutics
  • Ronald J. Bartek, President/Director/Co-founder, Friedreich's Ataxia Research Alliance
  • John J. Castellani, President & CEO, Pharmaceutical Research and Manufacturers of America (PhRMA)
  • Andrew J. Emmett, Managing Director, Science and Regulatory Affairs, Biotechnology Industry Organization
  • Thomas M. Burton, JD, Staff Reporter, The Wall Street Journal
  • Christopher Jennings, President, Jennings Policy Strategies
  • David P. Meeker, MD, President and CEO, Genzyme
  • Jose T. Morales, Partner, Accenture and Board Member, Cystinosis Research Network

Since 1983, NORD has been providing programs and services to improve the lives of all Americans affected by rare diseases.  It is a nonprofit organization providing advocacy, education and other services for patients and their families.  NORD's website is at

DIA is a nonprofit worldwide organization for professionals involved in the discovery, development and management of pharmaceuticals and medical devices.  Additional information about DIA may be found at

SOURCE National Organization for Rare Disorders (NORD)