From Voices to Breakthroughs: Celebrating the 2025 NORD Breakthrough Summit - National Organization for Rare Disorders Amid a government shutdown that has disrupted health care, slowed research, and strained complex systems, you came to the NORD Breakthrough Summit with

The future of #RareDisease care is here.

Curative therapies, AI-driven discovery, individually-tailored precision medicine, remote clinical trials, and equitable access models are no longer aspirations. They’re happening.

Read more in our 2025 #NORDSummit recap: rarediseases.org/2025-nord-br...

Thank you @rarediseaseadvisor.bsky.social for giving everyone a play-by-play of our 2025 #NORDSummit!

Read it for the latest in #RareDisease research and advocacy, orphan #pharmaceuticals, gene and cell therapy, and more.

Follow NORD for more updates like these year-round! @nordrare.bsky.social 🧡

NORD Rare Diseases & Orphan Products Breakthrough Summit 2025: #NORDSummit Discover video interviews, quotes, and insights from the 2025 NORD Breakthrough Summit—capturing innovation and advocacy in rare disease care.

🎥 Leaders from the #RareDisease community gathered in Washington, D.C. for the #NORDSummit @nordrare.bsky.social

We captured exclusive interviews, standout quotes, and insights shaping the future of research, advocacy & patient care

💬 Check out our full recap: https://bit.ly/4qwkvFc

Eli T. was honored to represent NBTS at this week’s 2025 NORD Summit in Washington, D.C., where experts and advocates convened to advance breakthroughs in research, treatments, and policies for the entire rare disease community.

#NORDSummit #BrainTumorResearch #RareDisease

#NORDSummit: Leah Howard, president of the National Psoriasis Foundation, speaking Oct. 21 at the @nordrare.bsky.social Breakthrough Summit in Washington, D.C.

Image & quote taken by Senior Correspondent, Larry Luxner.

#RareDisease #GeneralizedPustularPsoriasis #GPP #Psoriasis

#NORDSummit: Golie-Lorenzo Green, 45, of Houston, describing life with sickle cell disease. Green spoke Oct. 21 at the 2025 @nordrare.bsky.social Breakthrough Summit in Washington, D.C.

Image & quote taken by Senior Correspondent, Larry Luxner

#RareDisease #SickleCell #SCD #MedSky

That’s a wrap on the 2025 #NORDSummit — two incredible days of connection, collaboration, and commitment to shaping the future of #RareDisease research, treatment, and policy.

#NORD CEO Pam Gavin: “We're living in challenging times, but the rare community continues to show what unity looks like.” 💙

"I think about those kids right now who are trying their hardest despite the odds against them. And I want you to know you have changed lives. We have made progress, and I don't want to look at that progress in the rearview. We are moving forward together." - Senator Amy Klobuchar at the #NORDSummit

Senator Amy Klobuchar at the podium of the 2025 NORD Summit

Senator Amy Klobuchar at the podium of the 2025 NORD Summit

Senator Amy Klobuchar at the podium of the 2025 NORD Summit

Attendees and panelists applauding Senator Amy Klobuchar at the 2025 NORD Summit

U.S. Senator Amy Klobuchar, @klobuchar.senate.gov @amyklobuchar.com, Co-chair of the #RareDisease Congressional Caucus, surprised our #NORDSummit to underscore the bipartisan, national imperative to support rare patients right now, and to deliver treatments for the 95% of #RareDiseases without them.

#NORDSummit: Terence Flotte, MD, president of the 7,000-member @asgct.bsky.social, speaking Oct. 21 at the 2025 @nordrare.bsky.social Breakthrough Summit in Washington, D.C.

Image & quote taken by Senior Correspondent, Larry Luxner.

#RareDisease #MedSky #RareSky

#NORDSummit: Lee Greenwood, whose 5-year-old daughter Noa was born with #CanavanDisease, a severe #GeneticDisorder found mainly among Ashkenazi Jews. In June 2022, the girl received lifesaving #GeneTherapy and is now developing normally.
@nordrare.bsky.social

#RareDisease #RareSky #MedSky

A big moment for our team yesterday at #NORDSummit! 🦓 🌟 We shared two research posters, including our abstract on desmoid tumors, selected for the prestigious Lightning Round main stage! @nordrare.bsky.social

Dive into the full details below👇
🔗 bit.ly/42SXRfU
🔗 bit.ly/3Wi1o3B

#NORDSummit: @klobuchar.senate.gov (D-Minnesota), addressing the 2025 @nordrare.bsky.social Breakthrough Summit on Oct. 21 in Washington, D.C.

Image and quote taken by Senior Correspondent, Larry Luxner.

#RareDisease #MedSky #RareSky

#NORDSummit: Sarah Gheuens, MD, PhD, chief medical officer at Agios Pharmaceuticals, speaking Oct. 21 on #SickleCellDisease at the 2025 @nordrare.bsky.social Breakthrough Summit in Washington, D.C.

Image & quote taken by Senior Correspondent, Larry Luxner

#RareDisease #SickleCell #SCD #MedSky

#NORDSummit: Shelley Bowen, director of family services and advocacy for the Barth Syndrome Foundation, which successfully lobbied for FDA approval of #Elamipretide for an X-linked disease that affects only 500 boys worldwide. @nordrare.bsky.social

#RareDisease #BarthSyndrome #MedSky

#NORDSummit: Jackie Weinrich, senior health policy advisor to Rep. @dorismatsui.bsky.social (D-California), speaking Oct. 21 at the 2025 @nordrare.bsky.social Breakthrough Summit in Washington, D.C.

Image & quote taken by Senior Correspondent, Larry Luxner

#RareDisease #MedSky #HealthcarePolicy

#NORDSummit: Jana Monaco, NORD state ambassador for Virginia and mother of 2 children—Stephen and Caroline—with #IsovalericAcademia, a #MetabolicDisorder. Monaco spoke Oct. 20 at the 2025 @nordrare.bsky.social Breakthrough Summit.

Image & quote taken by Senior Correspondent, Larry Luxner

Proud to partner with #DyneTherapeutics and #EngageHealthInc at #NORDSummit 💙 Together we shared early findings from our Health Insurance Literacy in #myotonicDystrophy Type 1 (DM1) study—highlighting how collaboration helps move research forward and strengthens the #RareDisease community.

#NORDSummit: Ron Bartek, co-founder and president of the Friedreich’s Ataxia Research Alliance (FARA), speaking Oct. 20 at the 2025 @nordrare.bsky.social Breakthrough Summit

Image & quote taken by Senior Correspondent, Larry Luxner

#RareDisease #CellTherapy #GeneTherapy #FriedreichAtaxia

#NORDSummit: Rachel Sher, partner at Manatt Philips, and also former vice-president of policy at @nordrare.bsky.social, speaking Oct. 20 at the 2025 NORD Breakthrough Summit in Washington, D.C.

Image and quote taken by Senior Correspondent, Larry Luxner.

#RareDisease #MedSky #RareSky

#NORDSummit: Pamela Gavin, @nordrare.bsky.social CEO, speaking on the topic of affordability and accessibility for drugs during the C-suite Perspectives Session at the NORD 2025 Breakthrough Summit in Washington, D.C.

#RareDisease #HealthCare #MedSky #RareSky

#NORDSummit: Reenie McCarthy, CEO of Stealth BioTherapeutics, speaking Oct. 20, 2025, at the @nordrare.bsky.social 2025 Summit about the FDA’s Sept. 2025 approval of the Stealth therapy elamipretide (#Forzinity™) for #BarthSyndrome, an ultrarare, progressive genetic disease.

#NORDSummit: "Patient voice is so important...the risk is your risk, not our risk."

Catherine Owen-Adams, CEO of Acadia Pharmaceuticals speaking at the C-Suite Perspectives Session on Oct. 20, 2025, during @nordrare.bsky.social's 2025 Breakthrough Summit in Washington, D.C.

#RareDisease #MedSky

#NORDSummit: Emmelina Sophia Torres, 11, speaking about her #EDS diagnosis Oct. 20, 2025, during @nordrare.bsky.social's 2025 Breakthrough Summit in Washington, D.C.

Image & quote taken by Senior Correspondent, Larry Luxner

#RareDisease #EhlersDanlosSyndrome #EhlerDanlos #NORD #MedSky #RareSky

#NORDSummit: Pamela Gavin, CEO of @nordrare.bsky.social, speaking Oct. 20, 2025, at the opening session of NORD’s 2025 Breakthrough Summit in Washington, D.C.

Image & quote taken by Senior Correspondent, Larry Luxner

#RareDisease #MedSky #RareSky

Honored to be the only organization featured in Lightning Round presentations for 3 consecutive years at #NORDSummit! ✨ @nordrare.bsky.social

Catch us today 10/20:
12-12:30 PM - Lightning Rounds
5:30 PM - Exhibit Hall and the “Sip in Science” Poster Hall Reception

🔗 bit.ly/42LJWZ1
🔗 bit.ly/4qhqHAA

We couldn't agree more. Shoutout to Avery, Emmelina, Christopher, and Micah! #NORDSummit #RareDisease

Learn how top #pharma executives and patient advocates are tackling the biggest challenges in #RareDisease care—and stay at the forefront of collaboration and innovation—by joining the #NORDSummit live from anywhere 🌍

Reserve your Virtual Pass: nordsummit.org

NORD Breakthrough Summit Features Bold New Ideas for the Future of Rare Disease Care, Policy and Research - National Organization for Rare Disorders The Lightning Rounds Poster Presentations, slated for Oct. 20, are a highlight of the annual NORD Breakthrough Summit. This year’s selections reflect urgent

Next week's #NORDSummit features an entire hall of cutting-edge research on #RareDisease care, patient engagement, and policy.

Virtual attendees can explore the virtual poster hall and connect one-on-one with presenters through the conference app! rarediseases.org/nord-breakth...

Alltrna CMO Dr. Nerissa Kreher will join leaders from across the #raredisease field for a panel titled, “𝘼 𝙉𝙚𝙬 𝙀𝙧𝙖 𝙛𝙤𝙧 𝙍𝙖𝙧𝙚 𝘿𝙞𝙨𝙚𝙖𝙨𝙚 𝘾𝙡𝙞𝙣𝙞𝙘𝙖𝙡 𝙏𝙧𝙞𝙖𝙡𝙨,” at the 𝗡𝗢𝗥𝗗 𝗥𝗮𝗿𝗲 𝗗𝗶𝘀𝗲𝗮𝘀𝗲𝘀 & 𝗢𝗿𝗽𝗵𝗮𝗻 𝗣𝗿𝗼𝗱𝘂𝗰𝘁𝘀 𝗕𝗿𝗲𝗮𝗸𝘁𝗵𝗿𝗼𝘂𝗴𝗵 𝗦𝘂𝗺𝗺𝗶𝘁 in Washington, D.C.

#NORDSummit: nordsummit.org

@nordrare.bsky.social