NORD to Celebrate 2018 Rare Impact Awards and 35th Anniversary

On May 17^th, the National Organization of Rare Disorders (NORD) will host its 2018 Rare Impact Awards in Washington, DC at the Andrew W. Mellon Auditorium.

At the event, NORD will not only recognize efforts made to help the 30 million Americans affected by rare diseases, but the leading independent nonprofit organization will also celebrate the 35^th anniversaries of both its inception and the Orphan Drug Act.

In an article, president and CEO of NORD, Peter L. Saltonstall, stated: “There are 1 in 10 Americans living with rare diseases and more than half are children. We are looking forward to showcasing the stories of those are who bravely working towards a future in which every one of those people has access to a treatment or a cure. The evening will be incredibly special because this year, we are also marking NORD’s 35th anniversary as well as that of the Orphan Drug Act.”

Additionally, awards will be presented a number of individuals and organizations to recognize their efforts within the rare disease community. Robert Campbell, Jr, MD, Director of the Center for Thoracic Insufficiency Syndrome (CTIS), an Attending Physician in the Division of Orthopaedic Surgery at Children’s Hospital of Philadelphia (CHOP) and notable inventor of the Vertical Expandable Prosthetic Titanium Rib (VEPTR) device, will receive the Lifetime Achievement Award.

Estelle Benson, Founder of GBS|CIDP Foundation International, will receive the Abbey S. Meyers Leadership Award on behalf of the organization whose commitment is to support those living with Guillain-Barre syndrome (GBS), chronic inflammatory demyelinating polyneuropathy (CIDP), and related syndromes. Richard Pazdur, MD, Director of the Oncology Center of Excellence, will receive the Public Health Leadership Award.

Rare Impact Awards will be given to 7 individuals for a number of milestones. Recipients will include Patrick Dunegan; Elisabeth Dykens, PhD; Maria Kefalas, PhD; the Honorable Frank Pallone, Jr, Congressman from New Jersey; Ellen Sigal, PhD; the Honorable Marcy Toepel, State Representative from Pennsylvania; and Chris Ulmer. Credentials for the honorees are listed below:

Patrick Dunegan:

• Established a non-profit organization to support families with gastroparesis in Louisville, Kentucky.
• Is NORD’s Rare Action Network™ Volunteer State Ambassador for the state of Kentucky.
• Proposed legislation for a Rare Disease Advisory Council in his state.
• Hosted a Rare Disease Day.
• Tripled the number of Kentucky Rare Action Network members in only five months.

Elisabeth Dykens, PhD:

• Long-standing leader in the field of intellectual and developmental disabilities.
• Regarded as national and international authority on behavioral characteristics.
• First researcher to describe cognitive profiles and compulsivity in Prader-Willi Syndrome (PWS) patients.
• Received Association of University Centers on Disabilities Outstanding Achievement Award in 2011.
• Director of the Vanderbilt Kennedy Center for Excellence in Developmental Disabilities.
• Established the Academy of Country Music (ACM) Lifting Lives Music Camp for People with Williams Syndrome.
• Board Member on the Special Olympics and on the Scientific Advisory Board of the Prader-Willi Syndrome Association.

Maria Kefalas, PhD:

• Established The Calliope Joy Foundation, a nonprofit for and raised $450,000, which was used to help establish the nation’s first Leukodystrophy Center of Excellence at CHOP in 2015.

The Honorable Frank Pallone, Jr, Congressman from New Jersey

• Helped lead biomedical research funding into legislation to support progress toward treatments for rare diseases.

Ellen Sigal, PhD:

• Founded Friends of Cancer Research, a forward-thinking policy and advocacy organization.
• Accelerated the development of life-saving treatments across many disease areas, such as policies that have enhanced the development of orphan products and treatments for rare diseases.
• Helped develop Breakthrough Therapy Designation through Friends of Cancer Research.
• Made developments in the Lung Cancer Master Protocol (LungMAP) through Friends of Cancer Research.
• Made developments in the Patient Experience Data provisions of 21st Century Cures and 21st Century Cures Act through Friends of Cancer Research.

The Honorable Marcy Toepel State Representative from Pennsylvania

• Drafted a bill that was signed into law that will provide much-needed support to the rare disease community.

Chris Ulmer:

• Led a global dialogue on neurodiversity.
• Wrote a series of story books (Special Books by Special Kids) about medically diverse children, many with rare diseases.
• Interviewed hundreds of medically diverse children, many with rare diseases.

The black-tie event will include a piano performance by Michael Smedley, a talented young pianist who lost his sight due to the rare genetic degenerative retinal disease CRB1, and a performance by The David Bach Consort.

If you are unable to attend the Rare Impact Awards, you can watch the event live on a special webcast using Facebook Live on