News from NORD

NORD Rare Diseases and Orphan Products Summit to Feature Speakers from FDA, NIH, and ACMG


 

References

FDA Commissioner Robert Califf, MD, will deliver the keynote address on the opening morning of the annual NORD Rare Diseases and Orphan Products Summit, which is scheduled for October 17–18 in Arlington, Virginia. Dr. Califf will be among more than 20 FDA speakers and several from NIH at the event, which draws together patient advocates as well as government, industry, and academic professionals working with rare diseases.

David Flannery, MD, Medical Director of the American College of Medical Genetics, will talk about “Telemedicine and Rare Diseases,” and will present a live telemedicine demo. In a session on genetic innovation, moderator Nora Yang, PhD, MBA, from the NIH, and panelists from GeneDx, Intellia Therapeutics, Spark Therapeutics, and the FDA will discuss gene-editing, gene-sequencing and gene therapy.

Other topics to be addressed include the crucial role of data in advancing diagnosis and clinical drug development, focus on pediatric diseases, and the challenge of access and reimbursement.

The Summit will include a poster session. Poster abstracts may be submitted by students as well as professionals. August 19th is the deadline for abstracts. Read more about poster submissions.

Recommended Reading

ACP Develops Toolkit for Transition From Pediatric to Adult Care
MDedge Family Medicine
Foundation Seeks Proposals to Improve Care of Children With Chronic and Complex Diseases
MDedge Family Medicine
HCU Network Australia Develops Q & A Video With Homocystinuria Expert
MDedge Family Medicine
Pitt Hopkins Scientific Symposium to Take Place in November
MDedge Family Medicine
Hemophilia drugs top Medicaid spending per prescription
MDedge Family Medicine
Children under 6 with factor XIII deficiency had no major bleeds with recombinant product
MDedge Family Medicine
Aluminum chloride still helps control hyperhidrosis
MDedge Family Medicine
Sickle cell trait raises exertional rhabdomyolysis risk
MDedge Family Medicine
European Commission’s proposed criteria for endocrine disruptors trigger multiple concerns
MDedge Family Medicine
Study evaluates Zika syndrome with joint contractures
MDedge Family Medicine