NORD Rare Disease Summit on Tap for Oct. 17-18 in DC

Resources, networking offered for AADC deficiency patients, caregivers

Mary Chapman avatar

by Mary Chapman |

Share this article:

Share article via email
NORD rare disease summit | AADC News | announcement illustration of person with megaphone

The NORD Rare Diseases & Orphan Products Breakthrough Summit, set for Oct. 17–18, will feature a range of speakers and discussions about issues affecting the more than 300 million people worldwide thought to have a rare disease — including those with aromatic l-amino acid decarboxylase (AADC) deficiency.

The annual event is back in person and will be held at the Marriott Marquis in Washington, D.C. Registration is now open for the rare disease summit.

For those who sign up by Sept. 2nd, pricing ranges from $349, for NORD patient members, to $2,199 for industry participants. All prices include meals.

“Join your fellow rare disease leaders from patient advocacy groups, government, industry, and academia for exclusive access to fresh insights, compelling connections, and expert resources,” NORD states on an event webpage.

Recommended Reading
AADC deficiency treatment | AADC News | illustration of adult and child hugging

New Treatments Needed to Improve Quality of Life in AADC

In addition to speakers, the meeting will feature work sessions, discussion groups, poster presentations, and networking opportunities.

The summit will focus on individuals and families, and how the larger rare disease community can work together to enhance the lives of those living with a rare disorder. To underscore that focal point, this year’s address, “Finding Our Community,” will feature three speakers, who are themselves either patients, caregivers, or advocates.

Other speakers will include experts in federal policy, rare disease researchers, organization leaders, physicians, members of academia, and pharmaceutical representatives, according to a press release. Representatives from the U.S. Food and Drug Administration (FDA) also will be on hand for sessions.

Discussions will center around emerging issues in rare diseases, including data sharing, accelerated treatment approval, the impact of patient voices at the FDA, patient-focused therapy development, reimbursement strategies for gene and cell strategies, and global and equitable access to diagnosis and healthcare services. Some 95% of the 7,000 known rare conditions have no authorized treatment.

Other discussion topics will include mental health and rare cancers, cultures of inclusion in the workforce, how to launch a rare disease advisory council, and launching a patient registry. Sessions also will focus on the National Institutes of Health’s Rare Diseases Clinical Research Network, patient input in clinical trial design, the role of advocacy groups, and streamlining diagnoses in rare disease.

Collaboration between patient organizations and industry partners, grant writing, pediatric clinical trials, and teaching undergraduate students about rare diseases will also be discussion topics.

Still more presentations will focus on strategies for enhancing diversity, equity, and inclusion in rare disease research; the gap between medical innovation and community healthcare; ethical issues related to treatment; gene therapy; and newborn screening and rare diseases.

NORD is a patient advocacy organization that works to help rare disease patients and the organizations that support them. Last year’s virtual event attracted nearly 1,000 registrants from 33 countries.