First Annual Rare Disease Heroes™ Recognition Gala Honors Groundbreaking Treatment, Research at Top Institutions

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Rare Disease Report® Event October 17 to Celebrate Hospitals, Physicians, Researchers

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“This gala is our way to show appreciation to those working on rare diseases at teaching and research." - Jim Radke, editorial vice president, Rare Disease Communications.

Rare Disease Communications is proud to announce the First Annual Rare Disease Heroes™ Recognition Gala honoring five health care institutions and their medical teams during a celebration to be held Monday, October 17 in Arlington, Va.

Rare Disease Communications created the Rare Disease Heroes™ award to recognize teaching and research hospitals and their teams for outstanding contributions in the rare disease community related to endocrinology, genetics, hematology, lysosomal storage disorders and neurology.

More than 30 million Americans are coping with diseases defined as rare by the National Institutes of Health based on a prevalence of fewer than 200,000 affected individuals in the U.S. Roughly 7,000 rare diseases have been identified. Obtaining information, treatment and research funding for such specific conditions can be daunting for patients, families and medical professionals.

“Rare Disease Communications understands the unique challenges associated with rare disease research,” said James Radke, editorial vice president for Rare Disease Communications. “This gala is our way to show appreciation to those working on rare diseases at teaching and research.”

Radke added, “We had 100 nominations submitted. We heard from patients, advocates, and other clinicians who provided their input on what hospital and medical team should be acknowledged. It is a testimony to our judges that they were able to narrow the list of winners down to five.”

“Some great nominees made it a tough choice for some categories,” said nomination judge Dawn Laney, M.S., C.G.C., C.C.R.C., director of the Emory Genetic Clinical Trial Center at Emory University.

The 2016 Rare Disease Heroes™ are dedicated to changing the way rare conditions are managed, advancing understanding of a rare disease, helping patients better manage their conditions, and finding more efficient ways to identify and diagnose rare diseases. The 2016 Rare Disease Heroes™ include:

Endocrinology: Cook Children’s and Paul S. Thornton, M.D.
Cook Children’s, in Fort Worth, Tex., is one of the nation’s leading integrated pediatric health care systems providing families access to top-ranked specialty programs and a network of specialists, pediatricians, clinics, a medical center and community program to meet the unique needs of their children. Dr. Thornton is being recognized for his efforts to improve the management of children with congenital hyperinsulinism.

Genetics: Duke University Medical Center and Dwight Koeberl, M.D., Ph.D.
Comprised of 2,000 faculty physicians and researchers, the Duke University School of Medicine, along with the Duke University School of Nursing and Duke University Health System, create Duke Health. Dr. Koeberl’s research focuses on development of new therapy for inherited disorders of metabolism, especially glycogen storage disease.

Hematology: Cohen Children’s Medical Center and Adrianna Vlachos, M.D., Jeffrey Lipton, M.D., Ph.D. and Evangelia Atsidaftos, M.A.
Cohen Children's Medical Center opened in 1983 as the New York metropolitan area's only hospital designed exclusively for children and now is the largest provider of pediatric health services in New York State, serving 1.8 million children in Brooklyn, Queens, Nassau and Suffolk counties. Doctors Vlachos and Lipton and Ms. Atsidaftos are honored for advancing the understanding and patient treatment for Diamond Blackfan anemia, a disorder of the bone marrow.

Lysosomal Storage Disorders: Duke University and Priya Kishnani, M.D., Ph.D.
The Division of Medical Genetics at Duke University Medical Center is internationally recognized for delivering the highest quality of comprehensive clinical genetics services by addressing all aspects of patient care for rare genetic and metabolic diseases. Dr. Kishnani, who leads the Duke Lysosomal Storage Disease treatment center, has changed the lives of thousands of patients through her research and care.

Neurology: NYU School of Medicine and Horacio Kaufmann, M.D.
The NYU Dysautonomia Center is the leading center in the world dealing with rare disorders of the autonomic nervous system (dysautonomia). Dr. Kaufmann’s team provides compassionate, world-class and cutting-edge care to patients and families.

Heroes were selected by a panel of leaders in the field of orphan drug development and research who serve on the Rare Disease Report® editorial advisory board. Judges reviewed nominations from 100 members of the rare-disease community, including patients, advocates, caregivers and clinicians.

Hero winner Priya Kishnani, M.D., division chief of Medical Genetics at Duke University Medical Center stated, “This is truly wonderful and a huge honor.”

Honorees, along with patients and caregivers who nominated them, will share remarks during the gala, which will feature MLD Foundation President and Board Chair Dean Suhr as master of ceremonies. The event will be held 6 - 8 p.m., October 17 in Arlington, Va. Tickets are free but registration is required at

“Rare Disease Communications is proud to be honoring these real-life heroes,” said Chris Davis, president of Rare Disease Communications. “This is, indeed, a rare opportunity to applaud the small victories that mean so much to patients and families.”

About Rare Disease Report®
Rare Disease Report® is a website and weekly e-newsletter published since 2013 by the Michael J. Hennessy Associates, Inc. providing an independent voice for the rare disease community. It strives to bring together medical, scientific, investment, regulatory and advocate professionals interested in rare diseases and orphan drugs. To learn more, visit

About Rare Disease Communications
Rare Disease Communications is a health care communications and media company focused on the rare disease community and dedicated to raising awareness of rare diseases and orphan drugs so that patients will be diagnosed and treated more efficiently. Rare Disease Communications is part of the Michael J. Hennessy Associates, Inc. family of businesses, based in Plainsboro, N.J. MJH is dedicated to improving quality of life through health care education, research and communication. To learn more, visit


Rare Disease Communications Media Contact:
Becky Taylor, becky(at)btaylorpa(dot)com, 609-240-6886

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Becky Taylor