Track: Orphan Product Development Track Co-Chairs: Industry Representatives
Track: Diagnosis and Patient Care Track Co-Chairs: NORD Rare Disease Centers of Excellence & Industry Representatives
Track: Regulatory Perspectives in Rare Disease Medical Product Development Track Co-Chairs: FDA Representatives
Track: Research & Drug Development Track Co-Chairs: PAG and Industry Representatives
Authors of the top 5 selected poster abstracts will share their key findings.
Artificial intelligence and other innovative use of data show promise for greatly reducing the diagnostic odyssey and supporting more personalized and effective care.
Visit the Poster Hall and Exhibit Hall
Genome editing is emerging as a promising treatment approach for rare diseases. A panel of experts will discuss the current state of the science of these technologies and the regulatory framework of the approval process as we move into this new therapeutic area.