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

Actemra may reduce scleroderma skin thickening
MDedge Dermatology
VIDEO: TNF inhibitors improved refractory skin disease in juvenile dermatomyositis
MDedge Dermatology
Midostaurin cut organ damage in systemic mastocytosis
MDedge Dermatology
Poor outcome for PBC patient with bullous pemphigoid
MDedge Dermatology
Zika Understanding Unfolds
MDedge Dermatology
Study tracks distant metastatic patterns of Merkel cell carcinoma
MDedge Dermatology
Study tracks distant metastatic patterns of Merkel cell carcinoma
MDedge Dermatology
Benefits, challenges emerge in evolution of rheumatology-dermatology clinics
MDedge Dermatology
Aluminum chloride still helps control hyperhidrosis
MDedge Dermatology
Study evaluates Zika syndrome with joint contractures
MDedge Dermatology