New Jersey Rare Disease Day
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
Michael Losow, Amicus Therapeutics, Moderator
Sarah Balog Leukemia Lymphoma Society
Bill Cummings, Rare Disease Advocate
Stephanie Fischer, Rare Disease Patient Advocate
Paul Melmeyer, NORD
Rebecca Perkins, BioNJ
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:
Julie Raskin, CHI Executive Director, Moderator
Susanne Fitch, M.B.A. Commercial Advisor Zealand Pharma
Dominique Goodson, SCD Patient Advocate
Anita Gupta, M.D. Anesthesiologist, Pain Management, Pharmacologist, Princeton U.
Michelle Hall, EB Patient Advocate
Katherine Lord, MD, CHOP
John Maslowski, President and CEO, Fibrocell