Join the 2026 NORD Breakthrough Summit, Oct. 25–27 in Washington, D.C.! ASGCT staff will be there to connect you with patient education resources and discuss the latest policies affecting rare disease research and individuals. Learn more ➡️ nordsummit.org
#NORDSummit #RareDisease
#NORDSummit #RareDisease
September 23, 2026 at 4:27 PM
Join the 2026 NORD Breakthrough Summit, Oct. 25–27 in Washington, D.C.! ASGCT staff will be there to connect you with patient education resources and discuss the latest policies affecting rare disease research and individuals. Learn more ➡️ nordsummit.org
#NORDSummit #RareDisease
#NORDSummit #RareDisease
Leaders from the NORD #RareDisease Centers of Excellence will take the stage at October's #NORDSummit to share how they're tackling the field’s most pressing challenges: shortening time to diagnosis, improving coordinated care and advancing innovative research. Learn more & join us at nordsummit.org
September 11, 2026 at 1:16 PM
Leaders from the NORD #RareDisease Centers of Excellence will take the stage at October's #NORDSummit to share how they're tackling the field’s most pressing challenges: shortening time to diagnosis, improving coordinated care and advancing innovative research. Learn more & join us at nordsummit.org
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...
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...
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
rarediseases.org
November 3, 2025 at 2:41 PM
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...
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 🧡
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 🧡
🎥 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
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
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.
www.linkedin.com
November 3, 2025 at 2:32 PM
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 🧡
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 🧡
This is Avery, a young woman I have been honored to care for. She recently met with Senator Collins staff to advocate for the Rare Disease Pediatric Review Voucher program reauthorization AND to push through a grant for MSMDS and other rare disease research grants!
www.youtube.com/shorts/Xzwsv...
www.youtube.com/shorts/Xzwsv...
Senator Collin’s Office! #capitol #politics #nordsummit #rarediseases
YouTube video by Avery Garrison
www.youtube.com
October 29, 2025 at 9:17 PM
This is Avery, a young woman I have been honored to care for. She recently met with Senator Collins staff to advocate for the Rare Disease Pediatric Review Voucher program reauthorization AND to push through a grant for MSMDS and other rare disease research grants!
www.youtube.com/shorts/Xzwsv...
www.youtube.com/shorts/Xzwsv...
🎥 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
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
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.
www.linkedin.com
October 24, 2025 at 6:34 PM
🎥 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
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 #BrainTumorResearch #RareDisease
October 22, 2025 at 4:47 PM
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 #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
Image & quote taken by Senior Correspondent, Larry Luxner.
#RareDisease #GeneralizedPustularPsoriasis #GPP #Psoriasis
October 22, 2025 at 3:00 PM
#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
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
Image & quote taken by Senior Correspondent, Larry Luxner
#RareDisease #SickleCell #SCD #MedSky
October 22, 2025 at 2:38 PM
#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
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.” 💙
#NORD CEO Pam Gavin: “We're living in challenging times, but the rare community continues to show what unity looks like.” 💙
October 21, 2025 at 10:44 PM
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.” 💙
#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
October 21, 2025 at 6:50 PM
"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
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.
October 21, 2025 at 6:50 PM
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
Image & quote taken by Senior Correspondent, Larry Luxner.
#RareDisease #MedSky #RareSky
October 21, 2025 at 6:38 PM
#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
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
@nordrare.bsky.social
#RareDisease #RareSky #MedSky
October 21, 2025 at 6:26 PM
#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
@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
Dive into the full details below👇
🔗 bit.ly/42SXRfU
🔗 bit.ly/3Wi1o3B
October 21, 2025 at 6:07 PM
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
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
Image and quote taken by Senior Correspondent, Larry Luxner.
#RareDisease #MedSky #RareSky
October 21, 2025 at 3:20 PM
#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
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
Image & quote taken by Senior Correspondent, Larry Luxner
#RareDisease #SickleCell #SCD #MedSky
October 21, 2025 at 3:00 PM
#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
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
#RareDisease #BarthSyndrome #MedSky
October 21, 2025 at 2:30 PM
#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
#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
Image & quote taken by Senior Correspondent, Larry Luxner
#RareDisease #MedSky #HealthcarePolicy
October 21, 2025 at 2:15 PM
#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
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
Image & quote taken by Senior Correspondent, Larry Luxner
October 21, 2025 at 2:00 PM
#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
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.
October 20, 2025 at 11:04 PM
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
Image & quote taken by Senior Correspondent, Larry Luxner
#RareDisease #CellTherapy #GeneTherapy #FriedreichAtaxia
October 20, 2025 at 4:00 PM
#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
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
Image and quote taken by Senior Correspondent, Larry Luxner.
#RareDisease #MedSky #RareSky
October 20, 2025 at 3:45 PM
#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
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
#RareDisease #HealthCare #MedSky #RareSky
October 20, 2025 at 3:30 PM
#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
#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.
October 20, 2025 at 3:15 PM
#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.