NORD + 47 other patient advocacy organizations have united to express concern about the #Medicaid work requirement rule issued this week. We strongly urge #CMS to define medical frailty in a way that protects coverage and continuity of care for #RareDisease patients: rarediseases.org/nord-joins-c...

If you hear one quote from the 2026 NORD #RareDisease Scientific Symposium, let it be this: "Less than 5% of rare diseases have an #FDA approved treatment. We want to get that number up to 25%.” - Dr. Annica Wayman, #NIH NCATS Read our recap + watch full sessions: rarediseases.org/2026-rare-di...

In her #NORDSymposium keynote, Deputy Director of #NIH NCATS Dr. Annica Wayman declared the administration's bold goal of dramatically increasing the number of #FDA approvals for #RareDiseases, from 5% with treatments today to 25%. NORD looks forward to collaborating to make this vision a reality!

Dr. Gwen Nichols of Blood Cancer United issued a powerful call for #AcuteMyeloidLeukemia research: "We’ve made huge strides in treating childhood #cancer, but children with #AML are being left behind. Their survival lags far behind other pediatric #leukemia, and that’s simply not acceptable."

In a #NORDSymposium panel on Alternatives to Placebos & RCTs, Dr. David Margolin of #uniQure emphasized the vital role organizations like NORD, with our IAMRARE.org program, play in generating longitudinal data through patient registries & natural history studies. This is where investment is needed.

During her #NORDSymposium keynote address, Acting Director of @fda.gov #CDER Dr. Tracy Beth Høeg referenced #FDA's new Plausible Mechanism Framework and said clarifying guidances are coming from #CBER, the FDA Center for Biologics Evaluation & Research. #PlausibleMechanism #Biologics #RareDisease

Are you tuning in to the NORD #RareDisease Scientific Symposium next Tuesday and Wednesday, April 14-15? There's still time to register to attend the 2026 #NORDSymposium virtually! Review the agenda topics, including keynote speakers from #FDA and #NIH, and register today at nordscience.org.

We’re excited to support our partner @nordrare.bsky.social's 2026 #NORDSymposium. Support rare disease research readiness, trial design, and progress for patients. Learn more here: nordscience.org

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

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

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

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

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

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

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

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!

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

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

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#NORDSummit#CanavanDisease#GeneticDisorder#GeneTherapy#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#NORDSummit Dive into the full details below👇 🔗 bit.ly/42SXRfU 🔗 bit.ly/3Wi1o3B

#NORDSummit: Jackie Weinrich, senior health policy advisor to Rep. @dorismatsui.bsky.social (D-California), speaking Oct. 21 at the 2025 @nordrare.bsky.social#NORDSummitBreakthrough Summit in Washington, D.C. Image & quote taken by Senior Correspondent, Larry Luxner #RareDisease #MedSky #HealthcarePolicy

#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#NORDSummit#Elamipretide #RareDisease #BarthSyndrome #MedSky

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

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...

Meet the patient advocacy leaders shaping the future of #RareDisease care. At the 2025 #NORD Breakthrough Summit, they'll discuss clinical trial innovation, pediatric R&D, collaboration models, and more. Register to hear their voices and their visions: bit.ly/4lSAkDq

New #CME on #GiantCellArteritis highlights the importance of early diagnosis and sustained remission to prevent blindness, stroke, and relapse. Including updates on imaging, diagnostics, and targeted biologics. Enroll for free at @medliveofficial : 🩺 bit.ly/46cSDML

🚨 Big news! Dr. Jay Bhattacharya, Director of the National Institutes of Health (NIH) will deliver a special address to the #RareDisease community at the 2025 NORD Breakthrough Summit, Oct. 19-21 in D.C. This is your chance to hear directly from #NIH leadership! Register to attend: bit.ly/4lSAkDq

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.

How can #AI advance patient-centered drug development? #RareDisease experts will tackle this at the 2025 NORD Rare Diseases & Orphan Products Breakthrough Summit this October during a session led by Tracey Sikora, VP of Research & Clinical Programs at #NORD. Learn more and RSVP: nordsummit.org

Media passes available! The #NORD Breakthrough Summit, Oct. 19-21 in D.C., offers direct access to #RareDisease experts, patient advocates, industry executives, regulatory leaders, and investors to explore the latest developments in rare science, policy, and more. rarediseases.org/nords-2025-r...

ACPMP is proud to fund two $50K Catalyst Research Grants through the @nordrare.bsky.social #RareDisease Research Program. These grants will support scientific and/or clinical research related to #AppendixCancer/PMP Apps due: October 12, 2025 Apply now: rarediseases.org/advancing......

On #NationalPatientAdvocacyDay, we celebrate every patient advocate. 💙 Whether dedicating a career or rising to an unexpected challenge, your courage, persistence, and heart drive change. Thank you for using your voice to uplift, fight, and lead the #RareDisease community. ✨ #ThankYouAdvocates

The landscape of #RareDisease diagnosis and treatment is rapidly changing. What do clinicians, health care administrators, insurers, and others working in #medicine need to know? Find out at the 2025 NORD Breakthrough Summit. RSVP for the #NORDSummit now: nordsummit.org

The National Organization for Rare Disorders (NORD) is excited to attend the @ncslorg.bsky.social 2025 Legislative Summit in Boston next week to advocate for critical state #RareDisease policies. If you’re attending, visit us in the #NCSLsummit exhibit hall! Details: www.ncsl.org/events/2025-...