NORD, BioNJ and Patient Advocacy Groups to Collaborate on Rare Disease Day
Event at New Jersey State House
TRENTON, N.J. -- February 13, 2013
The National Organization for Rare Disorders, BioNJ and 11 New Jersey-based
patient advocacy groups are joining forces for a unique educational event
about rare diseases to be held at the New Jersey State House on March 4, 2013.
The gathering, which will convene local leaders from government, non-profit,
and the biotechnology sector, will be conducted on behalf of state residents
living with rare diseases that plague nearly 1 in every 10 Americans. The
event is highlighted by a New Jersey success story – the latest in-state
biopharmaceutical company to offer a significant treatment advance for one
group of patients with a rare intestinal disorder.
Patients, researchers, health care advocates, members of the New Jersey
government and biotech industry, as well as concerned citizens are invited to
attend the event on Monday, March 4, 2013, from 11:30 a.m. to 12:30 p.m. at
Committee Room 11, 4th floor, Annex Building, New Jersey Statehouse.
A wide variety of speakers will include researchers, clinicians, patients,
patient advocates and members of industry, including:
*Francois Nader, MD, President and CEO of NPS Pharmaceuticals of
Bedminster, NJ. In December, NPS received approval from the U.S. Food and
Drug Administration (FDA) for Gattex® (teduglutide [rDNA origin]) for
injection as a treatment for adult patients with short bowel syndrome who
are dependent on parenteral support. Dr. Nader was elected Chairman of the
BioNJ Board of Trustees earlier this month.
*Debbie Hart, President and CEO of BioNJ
*Mary Cobb, Senior Vice President, Membership at NORD
*Ian Marshall, M.D., pediatric endocrinology specialist at Robert Wood
Johnson University Hospital in New Brunswick, NJ.
*Ellen Welch, Director of Biologics (Genetic Disorders) at PTC Therapeutics
of Plainfield, NJ.
*Diana Autin, Executive Co-Director of the Statewide Patient Advocacy
Network (SPAN) of New Jersey
New Jersey State Senator Anthony Bucco (R-25) will also offer a resolution
designating the last day of February as Rare Disease Day and honoring all New
Jersey rare disease organizations.
The purpose of this event is to deliver the message that every patient counts
and that all patients, even those suffering from rare disorders, need
excellent health care and services to ensure a high quality of life. In the
United States, a disease is considered rare if it affects fewer than 200,000
people. There are more than 7,000 rare diseases affecting more than 30 million
people in the U.S. alone.
This event is one of many being held in honor of Rare Disease Day, an
international advocacy day bringing widespread recognition of rare diseases as
a global health challenge. NORD is the leader of rare disease activities in
the U.S. and is coordinating events at state houses across the country.
In addition to NORD and BioNJ, participating patient organizations include:
• Soft Bones
• Congenital Hyperinsulinism International
• CARES Foundation
• NOMID Alliance
• The Sturge Weber Foundation
• APS Type 1
• The Mastocytosis Society
• Children's Cardiomyopathy Foundation
• “Let Them Be Little X2"
• MDS Foundation
The National Organization for Rare Disorders (NORD), a 501(c)(3) organization,
is a unique federation of voluntary health organizations dedicated to helping
people with rare "orphan" diseases and assisting the organizations that serve
them. NORD is committed to the identification, treatment, and cure of rare
disorders through programs of education, advocacy, research, and service. NORD
serves all stakeholders in the rare disease community, including patients and
their families, patient organizations, researchers, medical professionals, and
companies developing orphan products. NORD works closely with many government
agencies, most notably the National Institutes of Health (NIH) and the Food
and Drug Administration (FDA). All NORD programs are focused on one ultimate
goal--to improve the lives of individuals and families affected by rare
BioNJ is singularly focused on advancing the growth and prosperity of New
Jersey’s biotechnology cluster through advocacy initiatives directed at State
and federal public policy issues and networking and educational programs that
bring the community together. Founded in 1994 by New Jersey industry CEOs, the
300-member organization serves as the voice of biotechnology companies located
in New Jersey, seeks to promote their economic growth and development and
works to encourage new and established companies from around the world to
locate to New Jersey. BioNJ represents companies engaged in biopharmaceutical,
biomedical, bioagricultural and bioremedial endeavors. To learn more about
BioNJ, please go to www.BioNJ.org.
Congenital Hyperinsulinism International
Bill O’Donnell, VP, Public Affairs and Communications
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