Lightning round poster presentations highlight cutting-edge insights on issues ranging from Medicaid coverage disparities to real-world data innovation
WASHINGTON, Oct. 13, 2025 /PRNewswire/ — The future of rare disease advancements will take center stage at the National Organization for Rare Disorders (NORD®) Rare Disease and Orphan Products Breakthrough Summit®, Oct. 19-21 in Washington.
NORD is proud to announce the top researchers selected for the Lightning Rounds Poster Presentations session. Chosen from a competitive field of submissions by academics, researchers, industry, government agencies, health care professionals, patient organizations, and students, the winning posters represent the forefront of innovation and advocacy in the rare disease space.
The Lightning Rounds Poster Presentations, slated for Oct. 20, are a highlight of the annual NORD Breakthrough Summit. This year’s selections reflect urgent and evolving priorities for the rare disease community, from addressing systemic barriers to genetic testing and harnessing real-world data for faster therapeutic discovery to creating collaborative care models that place patients at the center of innovation.
“These seven posters demonstrate what’s possible when research, policy, and advocacy come together with a shared commitment to innovation and patient empowerment,” said Pamela K. Gavin, NORD Chief Executive Officer. “We’re proud to showcase such forward-thinking work at the NORD Breakthrough Summit and look forward to seeing how these insights drive collaboration and change for the rare disease community.”
The winning posters and their authors are:
- Partnering for Education at a Turning Point in the Desmoid Tumor Landscape
(Presenting Author: Tariqa Ackbarali, PhD, CHCP, Medlive)
This case study explores the power of partnership through a collaboration among Medlive, NORD, and the Desmoid Tumor Research Foundation to provide free, accredited, digital education. The outcomes revealed the impact on clinical teams, improving their ability to diagnose and treat desmoid tumors. - The Castleman Disease Expert Panel: A Collaborative Model That Could Be Replicated to Improve Clinical Outcomes for Rare Disease Patients (Presenting Author: Madison Ahearn, BS, MHA, Castleman Disease Collaborative Network)
This presentation explores how an expert panel was developed to assist physicians in improving diagnosis, treatment, and overall patient care for Castleman disease through open, discussion-based reviews of complex cases, while demonstrating a scalable model to improve outcomes across other rare diseases. - Accelerating Rare Disease Discovery: Leveraging Real-World Data for Phenotypic Characterization and Therapeutic Target Identification (Presenting Author: Sanjay Ahuja, PhD, Regal Intel)
Real-world data captured via electronic health records, patient registries, insurance claims, and more holds the key to accelerating the discovery and development of much-needed orphan products, this study concludes. - Medicaid Coverage Gaps in Genetic Testing Lead to Health Disparities for Rare Disease (Presenting Author: Shannon Belmont, MPH, JM, GeneDx)
This study reveals a “significant and persistent disparity” in access to outpatient exome and genome sequencing between individuals with commercial insurance and those covered by Medicaid. Since rare disease diagnoses already take years, these gaps may further delay diagnosis and treatment for medically underserved and vulnerable populations. - Streamlining Patient Engagement in Rare Disease: A Multi-Sponsor Advisory Panel Pilot (Presenting Author: Shana Dodge, PhD, The Association for Frontotemporal Degeneration)
This pilot program brought together multiple companies to co-sponsor an advisory panel led by a patient advocacy organization, designed to efficiently gather insights from people affected by frontotemporal degeneration (FTD). The initiative streamlines input from a small patient population, reduces burden on individuals, and ensures clinical research reflects the real-world priorities of those living with FTD. - Quantifying Health Benefits of Orphan Drugs (Presenting Author: Genevieve Lyons, MSc, Alexion, AstraZeneca Rare Disease)
This study finds that orphan drugs, especially those with single or multiple rare disease indications, offer significantly greater health benefits than non-orphan drugs, highlighting the importance of informed policymaking that incentivizes scientific innovation. - Spotlight on Familial Chylomicronemia Syndrome (FCS): Leveraging the Power of Individual Journeys Toward Collective Action—Voice of the Patient Paper (Presenting Author: Alexandra Roeser, Arrowhead Pharmaceuticals)
To better understand the lived experience of those affected by familial chylomicronemia syndrome (FCS), Arrowhead Pharmaceuticals hosted a series of patient and caregiver convenings. Key insights highlight urgent needs for greater provider awareness and offer actionable strategies to improve diagnosis, care, and advocacy.
The Lightning Rounds Presentations, scheduled for Oct. 20 from 12-12:30 p.m. ET, is a dynamic, rapid-fire format designed to showcase the most compelling poster presentations in under five minutes each. This session is a cornerstone of the annual NORD Breakthrough Summit, offering visibility to rising voices and game-changing ideas across the rare disease space.
The NORD Breakthrough Summit, taking place at the Grand Hyatt in Washington, is an annual event dedicated to accelerating progress for the over 30 million Americans living with rare diseases.
This year’s theme, “From Voices to Breakthroughs,” underscores the power of lived experience in shaping scientific and policy progress. Featured speakers include patient advocates, industry executives, researchers at the forefront of scientific innovation, and regulatory leaders.
Learn more: https://nordsummit.org/
Media Registration:
If you are a member of an accredited media organization, please contact [email protected] to register for the NORD Breakthrough Summit.
About the National Organization for Rare Disorders
Founded in 1983, the National Organization for Rare Disorders (NORD®) is a leading independent, nonpartisan, nonprofit and patient advocacy organization dedicated to improving the health and lives of over 30 million Americans living with rare diseases. In partnership with more than 350 disease-specific member patient organizations, NORD drives progress in rare disease research, care, and policy. Learn more at https://rarediseases.org/
SOURCE National Organization for Rare Disorders (NORD®)
