NJRareDiseaseAlliance

For more information, please contact: eszoke@sculptahealth.com



New Jersey Rare Disease Day Event

Monday,  March 5th, 2018


Event Location: 

NJ State Museum

Riverside Court (1st Floor)

205 West State Street 

Trenton, NJ


A Rare Disease Day Event in Trenton

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


The event will take place at the NJ State  Museum, Riverside Court (1st Floor), 205 West State Street, Trenton, NJ.

The day’s events are being sponsored by BioNJ,  HINJ,  and NORD.

 Event Schedule
Final speakers and times subject to change


   8:30 - 9:00       Continental Breakfast and 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


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

 ​*NORD (The National Organization for Rare Disorders) is the official sponsor of Rare Disease Day in the US. They have inspired advocates around the country to hold events in their state capitols. Last year there were 32 State House events across the United States.  State House Events provide an opportunity to meet face-to-face with your elected officials and advocate for legislation that directly affects the rare disease community.​