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
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...
We couldn't agree more. Shoutout to Avery, Emmelina, Christopher, and Micah! #NORDSummit #RareDisease
October 20, 2025 at 1:54 PM
We couldn't agree more. Shoutout to Avery, Emmelina, Christopher, and Micah! #NORDSummit #RareDisease
#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: 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
#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
#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
Image & quote taken by Senior Correspondent, Larry Luxner
#RareDisease #MedSky #RareSky
October 20, 2025 at 2:36 PM
#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
Image & quote taken by Senior Correspondent, Larry Luxner
#RareDisease #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.
#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
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.” 💙
#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: 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
Image & quote taken by Senior Correspondent, Larry Luxner
#RareDisease #EhlersDanlosSyndrome #EhlerDanlos #NORD #MedSky #RareSky
October 20, 2025 at 2:50 PM
#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
Image & quote taken by Senior Correspondent, Larry Luxner
#RareDisease #EhlersDanlosSyndrome #EhlerDanlos #NORD #MedSky #RareSky
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 🧡
#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: 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
📣 Medlive will be on-site for the 2025 NORD® #RareDiseases and Orphan Products Breakthrough Summit® in D.C.!
We are honored to be the only organization featured in Lightning Round poster presentations for three consecutive years, from 2023–2025. 🔗 bit.ly/3KypyEx
@nordrare.bsky.social #NORDSummit#NORDSummit
We are honored to be the only organization featured in Lightning Round poster presentations for three consecutive years, from 2023–2025. 🔗 bit.ly/3KypyEx
@nordrare.bsky.social #NORDSummit#NORDSummit
October 6, 2025 at 2:12 PM
📣 Medlive will be on-site for the 2025 NORD® #RareDiseases and Orphan Products Breakthrough Summit® in D.C.!
We are honored to be the only organization featured in Lightning Round poster presentations for three consecutive years, from 2023–2025. 🔗 bit.ly/3KypyEx
@nordrare.bsky.social #NORDSummit#NORDSummit
We are honored to be the only organization featured in Lightning Round poster presentations for three consecutive years, from 2023–2025. 🔗 bit.ly/3KypyEx
@nordrare.bsky.social #NORDSummit#NORDSummit
"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
🎥 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
Final week of #NORDSummit early bird pricing!
Save hundreds when you attend the most anticipated #RareDisease conference for the #pharmaceutical, #biotech, and #MedicalDevice industries.
Register your team for the NORD Rare Diseases & Orphan Products Breakthrough Summit today at nordsummit.org.
Save hundreds when you attend the most anticipated #RareDisease conference for the #pharmaceutical, #biotech, and #MedicalDevice industries.
Register your team for the NORD Rare Diseases & Orphan Products Breakthrough Summit today at nordsummit.org.
September 4, 2025 at 3:01 PM
Final week of #NORDSummit early bird pricing!
Save hundreds when you attend the most anticipated #RareDisease conference for the #pharmaceutical, #biotech, and #MedicalDevice industries.
Register your team for the NORD Rare Diseases & Orphan Products Breakthrough Summit today at nordsummit.org.
Save hundreds when you attend the most anticipated #RareDisease conference for the #pharmaceutical, #biotech, and #MedicalDevice industries.
Register your team for the NORD Rare Diseases & Orphan Products Breakthrough Summit today at nordsummit.org.
🚨 Attention #PublicHealth and #RareDisease Researchers: Share your groundbreaking work at the 2025 #NORDSummit!
Submit your abstract by midnight, July 18 for display in the Poster Hall + a chance to present your #research on the main stage.
Check out the categories and apply now: bit.ly/3LiYAw8
Submit your abstract by midnight, July 18 for display in the Poster Hall + a chance to present your #research on the main stage.
Check out the categories and apply now: bit.ly/3LiYAw8
June 18, 2025 at 4:17 PM
🚨 Attention #PublicHealth and #RareDisease Researchers: Share your groundbreaking work at the 2025 #NORDSummit!
Submit your abstract by midnight, July 18 for display in the Poster Hall + a chance to present your #research on the main stage.
Check out the categories and apply now: bit.ly/3LiYAw8
Submit your abstract by midnight, July 18 for display in the Poster Hall + a chance to present your #research on the main stage.
Check out the categories and apply now: bit.ly/3LiYAw8
#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: 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: 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: 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
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
Reserve your Virtual Pass: nordsummit.org
October 17, 2025 at 2:14 PM
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
Reserve your Virtual Pass: nordsummit.org