National Organization for Rare Disorders (NORD)

This image features a quote from Edward Neilan, Chief Medical and Scientific Officer at NORD, discussing the importance of relevant and reliable real-world data for regulators like the FDA. It mentions AI's potential role in processing large data amounts. The background is a light blue graphic design. The bottom right corner displays the Rare Disease Advisor logo.

#MDAConference: Edward Neilan, Chief Medical and Scientific Officer at @nordrare.bsky.social, speaking at the @mda.org's 2026 Clinical & Scientific Conference in Orlando, FL.

#RareDisease #MuscularDystrophy #AI #MedTech

Happy #RareDiseaseDay! Your first as a #NORD Rare Disease Center of Excellence, earned through years of investment and dedication to rare patients. We're thrilled to celebrate many more Rare Disease Days together.

More info on our NORD Centers of Excellence Network: rarediseases.org/center-of-ex...

More great #RareDiseaseDay content from our #NORD Rare Disease Centers of Excellence! This work happens all year long in #Colorado and all across America.

Learn about the NORD Rare Disease Centers of Excellence network and find a Center near you: rarediseases.org/center-of-ex...

The Division of Medical Genetics prepared for #RareDiseaseDay with an outreach event, complete with many zebras, at Emory University Hospital. Thanks especially to Dr Rossana Sanchez Russo for organizing! @emoryhealthcare.bsky.social

Two people stand on the left of a table and two people stand on the right of it. On the table is a trifold with information about Rare Disease Day as well as giveaways that include squishy zebras.

A close-up of the back of a squishy zebra that says "NORD Rare Disease Center of Excellence" on it.

Three people stand talking: Dr. Vivian on the left, Erika with Children's Mercy's Rare PFAC in the middle, and a mother of a kiddo in our NICU on the right.

A woman picks an item up off of the giveaway table while smiling.

This week @jaylvivian.bsky.social spent time with members of @childrensmercy.org's Rare PFAC tabling at the hospital. Besides resources and giveaways, Dr. Vivian also talked about our NORD Rare Disease Centers of Excellence designation. #RareDisease #RareDiseaseDay

Thank you @pearljam.com for raising awareness for rare diseases like EB, epidermolysis bullosa, this #RareDiseaseDay! We're inspired by your advocacy.

Our team at @nordrare.bsky.social has got your back today and every day. Let's work together to support all #rare families.

➡️ rarediseaseday.us

#RareDiseases affect 1 in 10 patients, but medical training on them is limited. We can change that.

Join the #RareDisease #CME Challenge with NORD and
@medliveofficial.bsky.social
this #RareDiseaseDay and earn your stripes to better support your rare patients: medlive.com/nord

Rare is EVERYWHERE. Show Your Stripes for the 1 in 10 Americans living with a rare disease. Global Rare Disease Day, February 28.

1 in 10 Americans—our friends, neighbors, coworkers, classmates, and loved ones—is living with a #RareDisease.

That's 30 million+ people.

Follow us, the National Organization for Rare Disorders, #NORD, to support them this #RareDiseaseDay.

Learn more at rarediseaseday.us.

We are proud to launch the first Earn Your Stripes: Rare Disease CME Challenge exclusively in partnership with @nordrare.bsky.social improve the early identification, diagnosis, and management of rare conditions. Start earning your stripes now: bit.ly/4rDTyQ4

The average rare-disease patient waits years for a diagnosis, often seeing multiple specialists before receiving answers. It’s time to change that.

Join the Rare Disease CME Challenge with @nordrare.bsky.social to get started: bit.ly/4kVUgph

Graphic featuring a child and doctor that reads, "On behalf of NORD and our 46 Rare Disease Centers of Excellence, Thank You, Pediatricians! Happy National Pediatrician Day.

This #NationalPediatricianDay, we extend a special shoutout to the best-in-class #pediatricians at our 46 NORD Rare Disease Centers of Excellence!

>15 million children in America have a #RareDisease. As a #pediatrician, every day you work, you are at the front line of rare diagnosis and treatment.

Hey #creators, #gamers, and #streamers: Join #StreamingForRare on Feb. 28 for #RareDiseaseDay! 🎮

Register your stream, go live with content you love, and invite your audience to donate to the #RareDisease community. Sign up here: bit.ly/4qLKTuk

Twilight view of the Cedars-Sinai Medical Center with illuminated streets and light trails from moving vehicles.

Cedars-Sinai has been named a Rare Disease Center of Excellence by @nordrare.bsky.social

The designation recognizes expertise in rare disease research, diagnosis and specialized care and connects Cedars-Sinai with a national network of 46 institutions.

Learn more: https://ceda.rs/4rZCtAw

CNBC launches CNBC Cures to help 30 million Americans with rare diseases CNBC Cures aims to raise awareness of rare diseases. Led by "Squawk Box" anchor Becky Quick, CNBC Cures was inspired by her family's own rare disease journey.

NORD applauds @cnbc.com for launching CNBC Cures, using its influential business platform to confront one of the most urgent public health challenges of our time: 95% of 10,000+ known #RareDiseases still lack an FDA-approved treatment. The time for investment is now.
www.cnbc.com/2026/01/08/c...

📣 Medlive & @nordrare.bsky.social (NORD) have extended our exclusive CME partnership. We are eager to introduce innovative education models, integrate real-world data, and continue to empower the #RareDisease community. Read the full press release here: bit.ly/3YuWGka

CNBC's Becky Quick details daughter's rare disease journey CNBC Squawk Box anchor Becky Quick details her daughter's rare disease journey, and explains why it inspired her to launch CNBC Cures.

CNBC's Becky Quick details daughter's rare disease journey

NORD Scientific Symposium - Advancing Innovation in Rare Disease Research Rare disease breakthroughs require collaboration. Join a community of researchers, clinicians, industry leaders, and others at the NORD® Rare Disease Scientific Symposium to share knowledge, exchange ...

Join us in celebrating this news with the teams at @childrensphila.bsky.social & @pennmedicine.bsky.social, a #NORD Rare Disease Center of Excellence, who carried out this historic procedure. They spoke at our 2025 Rare Disease Scientific Symposium, and registration is open for 2026! nordscience.org

First Baby to Undergo Gene Editing Takes First Steps: ‘It’s All Been a Miracle’ The first infant to undergo gene-editing therapy has accomplished another first — his first steps. KJ Muldoon is walking and getting ready to spend Christmas at home after being hospitalized last holi...

Baby KJ, the first person ever to undergo personalized #GeneEditing, took his first steps! ❤️

Babies with #CPS1 have a 50% chance of living past one week. This week, thanks to years of #RareDisease research, investment and advocacy, KJ will celebrate his second Christmas. people.com/first-baby-t...

Feb 28 is #RareDiseaseDay! Help raise awareness by lighting up local buildings and monuments in the official colors.

💡 You can find instructions, templates, and more here:
rarediseases.org/rare-disease...

Tag @RareDiseases and use #LightUpForRare when you post your photos on Feb 28!

Nighttime view of the Children's Mercy Hospital building, illuminated and displaying the logo for the NORD Rare Disease Center of Excellence.

We’re proud to be named a NORD® Rare Disease Center of Excellence, a network of 46 institutions nationwide advancing expert care + research for rare disease patients. https://cmkc.link/3Y64yZ0 @nordrare.bsky.social #RareDisease

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 🧡

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.” 💙

#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

#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: @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

"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: 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