NORD Rare Diseases and Orphan Products Breakthrough Summit

Guest post by NORD

The challenges of providing care for patients with chronic and complex rare diseases will be a major topic at the upcoming NORD Rare Diseases and Orphan Products Breakthrough Summit, to take place Oct. 21 – 22 in Arlington, VA. This annual conference draws together medical professionals, patient advocates and thought leaders from government and industry to address collectively issues related to rare diseases and orphan therapies.

NORD – the National Organization for Rare Disorders – is a nonprofit organization that since 1983 has been the voice of the rare disease community. With programs of advocacy, education, research and patient assistance, NORD connects patients with those seeking to improve their lives.

The 2016 Breakthrough Summit will feature sessions on promoting early diagnosis, newborn screening, precision medicine and optimizing clinical trial success, among many other topics. Keynote speakers will include Stephen Ostroff, MD, acting commissioner of the U.S. Food and Drug Administration (FDA), and Christopher P. Austin, MD, director of the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH).

The opening session will feature Robert Saul, MD, one of the co-directors of the recently completed American Academy of Pediatrics (AAP) three-year Genetics in Primary Care initiative. Dr. Saul will discuss what was learned regarding the needs of community physicians who have children with complex genetic diseases in their practice.

In a session on precision medicine, Barbara Conley, MD, associate director of the Cancer Diagnosis Program at the National Cancer Institute (NCI), will provide an overview of the NCI-Match clinical trial launched earlier this year. This major nationwide study will explore whether cancer therapies targeted to specific gene mutations may be effective regardless of the type of cancer.   

Other speakers will include Mike S. Watson, PhD, executive director of the American College of Medical Genetics and Genomics, who will address the topic, “Newborn Screening – Advanced Capabilities and Ethical Considerations.”

The NORD Breakthrough Summit is the only rare disease conference co-sponsored by the FDA, and it includes – in addition to general sessions – special tracks for those developing treatments, those providing medical care and those advocating on behalf of patients. Last year, nearly 500 participants attended the Summit, including leaders of approximately 80 rare disease patient organizations.
The Summit is open to all. Information about the program, registration and sponsorship is available on the NORD website
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