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New Jersey Rare Disease Day

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March 05, 2018
8:30 am - 3:30 pm

New Jersey Museum


A Better World for People Living with Rare Diseases: State, National, and Global Action


Sponsored by, BioNJ, HINJ and NORD

Takes place at the New Jersey Museum at 205 West State Street, in Trenton.

From 8:30 a.m. – 3:30 p.m. on March 5.

AGENDA: (Final Speakers and Times, subject to change)

8:30 – 9:00 Continental Breakfast and open networking.

9:00 – 9:05 Opening Remarks, Ellynn Szoke, NJ Rare Disease Alliance

9:05 – 9:55 Effective Advocacy Panel and Q&A – Real World Advice

  1. Michael Losow, Amicus Therapeutics, Moderator

  2. Sarah Balog Leukemia Lymphoma Society

  3. Bill Cummings, Rare Disease Advocate

  4. Stephanie Fischer, Rare Disease Patient Advocate

  5. Paul Melmeyer, NORD

  6. Rebecca Perkins, BioNJ

  7. Hemophilia Treatment Center/Distribution Company representative, TBD

9:55 – 10:00 Speaker Introduction by Dean J. Paranicas, President & CEO, HINJ

10:00 – 10:30 Keynote Speaker and Q&A Congressman Leonard Lance (R – NJ Dist. 7)

10:30 – 10:45 Break

10:45 – 10:50 Speaker Introduction by Debbie Hart, BioNJ President &CEO

10:50- 11:15 NJ Dept. of Health, Office of Commissioner and Q&A

11:15 – 12:00 Clinical Development Panel Discussion and Q&A, Panelists:

  1. Julie Raskin, CHI Executive Director, Moderator

  2. Susanne Fitch, M.B.A. Commercial Advisor Zealand Pharma

  3. Dominique Goodson, SCD Patient Advocate

  4. Anita Gupta, M.D. Anesthesiologist, Pain Management, Pharmacologist, Princeton U.

  5. Michelle Hall, EB Patient Advocate

  6. Katherine Lord, MD, CHOP

  7. John Maslowski, President and CEO, Fibrocell

12:00 – 12:30 Closing Remarks and Lunch, Ellynn Szoke, NJ Rare Disease Alliance

12:30 – 12:45 Walk to State House

12:45 – 3:30 State House Individual meetings with State Representatives

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