National Organization for Rare Disorders (NORD)
@RareDiseases
22m
25 runners. 26 #RareDisease community partners. More than $200,000 raised so far. Our hearts are full after the 2026 #NYCMarathon ❤️

This year, Team #RunningForRare was the largest in our history. Thank you to @nycmarathon, @nyrr, and everyone who donated and cheered us on.

You ence for people with #RareDiseases by reading our runners’ stories and supporting their fundraisers here: bit.ly/46IcZy0
0
0
0
81
National Organization for Rare Disorders (NORD)
@RareDiseases
1h
This #PulmonaryHypertension Awareness Month, learn about #PulmonaryArterialHypertension, a rare condition causing high blood pressure in the arteries of the lungs that can be difficult to recognize because early symptoms resemble other conditions.

Watch the full 6-minute video: youtube.com/watch?v=iIzY0X…

#PHAwarenessMonth #Pulmonology #Cardiology #PAH
0
0
0
117
National Organization for Rare Disorders (NORD)
@RareDiseases
5h
Caring for patients with #NF1-associated plexiform neurofibromas can be complex, especially during the transition to adult care.

Explore strategies for patient selection, adverse event management, and care coordination in this free, on-demand #CME program from NORD and ://t.co/AyoIoXGEwf

#MedicalEducation #MedEd #neurofibromatosis
A person is reading a piece of paper in an office setting. The image features a banner for the Clinician Education Program and highlights "Bridging Gaps Across the NF1-PN Care Continuum." It mentions the availability of on-demand content and includes logos for medlive, NORD (National Organization for Rare Disorders), and the Children's Tumor Foundation.
0
0
4
254
National Organization for Rare Disorders (NORD)
@RareDiseases
Oct 30
Run #Boston. Run for Rare. 🏃‍♀️💙

Applications are open for the 2027 #BostonMarathon with NORD #RunningForRare! Only 5 competitive team spots. Fundraise, advocate, and run with purpose. Apply by Nov. 1: runningforrare.org/events/

Boston is just the beginning! In 2027, run with NORD Marathon, @grandmasmara, @tokyo42195_org, or @runcolfax.

Every mile tells a story. Make yours one that moves the #RareDisease community forward.
1
0
4
337
National Organization for Rare Disorders (NORD)
@RareDiseases
Oct 30
Join @ASGCTherapy for Breakthroughs in Cell and #GeneTherapy, Nov. 12–13 in Nashville or online.

Cell and gene therapy is advancing rapidly, bringing new opportunities and challenges. Explore #CNS advances, targeted delivery, and manufacturing successes while connecting with field.

Register today: bit.ly/asgct-breakthr…
A cityscape of Nashville with the logo of the American Society of Gene and Cell Therapy. Text announces "Breakthroughs in Cell + Gene Therapy" taking place on November 12-13, 2026, in Nashville, TN and online.
1
1
4
351
National Organization for Rare Disorders (NORD)
@RareDiseases
Oct 29
Join the IPPF @healourskin for their next Patient Education Webinar on November 13 at 11 a.m. PT/ 2 p.m. ET, an open Q&A with an expert physician about living with #pemphigus and #pemphigoid.

RSVP: zoom.us/webinar/regist… #dermatology
0
0
2
361
National Organization for Rare Disorders (NORD)
@RareDiseases
Oct 29
A federal court sided with 25 states and D.C., striking down an added test for medically frail individuals under new #Medicaid work rules. The court also barred the federal government from penalizing those states for implementation errors or delays for six months.

#NORD is nd will continue our work to protect coverage for the #RareDisease community.

Read the court order: storage.courtlistener.com/recap/gov.usco…

#StayCovered #HR1 #WorkRequirements
Quote: “Access to care for the rare disease community has long been NORD’s priority. It is particularly important that eligible people do not lose coverage through implementation inconsistent with the intent of the law.” Attribution: Kathryn Lowell, Executive Vice President, Policy and Government Affairs, NORD.Map of 25 plaintiff states and D.C. that brought the case in front of Federal Court. Click link to read list of states.
0
1
3
324
National Organization for Rare Disorders (NORD)
@RareDiseases
Oct 29
Here are 2 ways your experience can build greater understanding of life with #POLG.

Complete the POLG survey THIS WEEK by October 31: survey.alchemer.com/s3/8950869/POL…

Then, join @MitoAction for the POLG Externally-led Patient-Focused Drug Development #ELPFDD Meeting on November 17, 10 a.m. ET. Register here: buff.ly/hgIWQuA

#MitochondrialDisease
0
0
1
328
National Organization for Rare Disorders (NORD)
@RareDiseases
Oct 28
Pam Gavin, President and CEO of the National Organization for Rare Disorders (#NORD), spoke on behalf of #RareDisease advocates at today’s @FDA Public Meeting on the Rare Disease Framework Initiative hosted by @reaganudall, offering strategic direction, partnership, and DA’s efforts.

We heard a few common themes rise up from the public comments about the importance of the patient voice, clarity and consistency, and ensuring the evidence requested by #FDA is reasonable and appropriate. These echoed this week's conversations at the #NORDSummit, and we're committed to addressing them together with urgency.
NORD President and CEO Pam Gavin addresses FDA Rare Disease Framework Initiative Public Meeting, October 28, 2026. Photo from event.
0
1
12
574
National Organization for Rare Disorders (NORD)
@RareDiseases
Oct 28
NORD's policy leaders appreciated the opportunity to have candid discussions on key #FDA modernization initiatives with the directors of each @FDA center both onstage and offstage at the 2026 #NORDSummit.

Pictured here with the Director of the Center for Drug Evaluation and ke Davis (center right) is #NORD EVP of Government Affairs Kathryn Lowell (center left), VP of Federal & Global Public Policy Mike Beard (right), and Policy Analyst Sarah Royka (left). @FDADrugs #RareDisease
Director of the FDA Center for Drug Evaluation and Research Dr. Mike Davis (center right), NORD Executive Vice President of Government Affairs Kathryn Lowell (center left), Vice President of Federal and Global Public Policy Mike Beard (right), and Policy Analyst Sarah Royka (left).
0
0
8
596
National Organization for Rare Disorders (NORD)
@RareDiseases
Oct 28
Advances in precision #oncology are creating new opportunities for people living with rare #cancer, but access to biomarker testing remains uneven in many states.

Yesterday's #NORDSummit panel on Closing the Gap in Precision Oncology with @ACSCAN, @ChildrensNatl, and NJ Senator J7) explored the policy solutions and patient advocacy needed to expand equitable access to testing.

#HealthEquity #biomarkers
0
2
10
584
National Organization for Rare Disorders (NORD)
@RareDiseases
Oct 27
Changes to #Medicaid are coming fast. Work requirements and accompanying eligibility questions, like the two-part test, are first.

#NORDSummit panelist Dr. Heather Saunders of @KFF says the impact could be felt hardest in #rural areas.

NORD has an FAQ and recommended policies nsure #RareDisease patients retain coverage: rarediseases.org/medicaid-work-…
The image is a bar graph showing the comparison between federal Medicaid spending reductions in rural areas and the rural health fund. The left bar indicates a reduction of approximately $137 billion in federal Medicaid spending, and the right bar shows a $50 billion rural health fund. Text notes that states' awards range from less than $100 per rural resident to more than $500. The image is attributed to KFF.
0
1
3
430
National Organization for Rare Disorders (NORD)
@RareDiseases
Oct 27
In our #NORDSummit panel, Closing the Access Gap in Precision Oncology, our panelists highlighted the power that #biomarker testing has for #cancer patients and how too many people lack access to it. The science is already here, the problem is affordability and access.

NJ State ined how #NewJersey passed a law requiring #HealthInsurance coverage for biomarker testing. This must be a #HealthEquity model for other states to follow, and about half of states so far have.

Thank you to our panelists from @ChildrensNatl, @ACSCAN and moderator Jim Palma of @TargetCancer
Quote from Hilary Gee Goeckner of the American Cancer Society Cancer Action Network at the 2026 NORD Breakthrough Summit: "Biomarker testing is the key that unlocks access to precision medicine: better understanding of your diagnosis, your prognosis, and what treatments are available. And unfortunately, we see a lot of the same disparities that we see across the healthcare system in access to biomarker testing."
2
3
12
598
National Organization for Rare Disorders (NORD) retweeted
U.S. FDA
@FDA
Oct 27
Modernizing FDA's review process starts with listening. Acting Commissioner Kyle Diamantas joined @RareDiseases at the #NORDSummit Monday to discuss how patient voices are central to building a fit-for-purpose framework for #RareDisease therapies.
National Organization for Rare Disorders (NORD)
@RareDiseases
Oct 26
Acting @FDA Commissioner Kyle Diamantas, @FDA_KyleD, sat down for a fireside chat with #NORD President and CEO Pam Gavin to kick off the 2026 #NORDSummit, where he shared that the #FDA is committed to modernizing its review process and is relying on #RareDisease patient voices to purpose regulatory framework. We appreciated the opportunity to break down what that will entail, and we're excited to be a partner in this crucial, timely mission.
Acting FDA Commissioner Kyle Diamantas speaking with NORD President and CEO Pam Gavin at the 2026 NORD Summit.
16
15
85
14,285
National Organization for Rare Disorders (NORD)
@RareDiseases
Oct 27
During our 2026 #NORDSummit FDA Rare Disease Regulatory Roundtable, moderator Amy Comstock Rick asked the Directors of #FDA CDER, CBER, and CDRH: Where can patient voices make the most difference?

Center for Devices and Radiological Health (#CDRH): Identifying unmet needs, ices, involving patients in design of clinical trials to maximize participation and efficacy, gauging product quality once released

Center for Biologics Evaluation and Research (#CBER): Risk-benefit analysis of #GeneTherapy, defining acceptable outcomes and meaningful clinical endpoints

Center for Drug Evaluation and Research (#CDER): Patient-focused drug development meetings help explain which symptoms needs to be treated and which do not
FDA Regulatory Roundtable at the NORD Breakthrough Summit 2026. From left to right, head of the Rare Disease Innovation Hub Amy Comstock Rick (moderator); Michael Davis, MD, PhD, Director, CDER; Michelle E. Tarver, MD, PhD, Director, CDRH; Karim Mikhail, B.Pharm, MSc, Director, CBER.
2
1
13
3,271
National Organization for Rare Disorders (NORD)
@RareDiseases
Oct 26
We're sharing today's full, hour-long #NORDSummit conversation between the CEOs of #NORD, @BioMarin, @JazzPharma, and @OtsukaUS, moderated by @CNBC Cures host @BeckyQuick, so that the entire rare disease community can hear their urgent insights about the next decade of #RareDisease research and care. Watch it now: youtube.com/watch?v=UDf3BP…
0
3
25
6,411
National Organization for Rare Disorders (NORD)
@RareDiseases
Oct 26
We had the honor of presenting Mark Skinner with a Lifetime Achievement #RareImpactAward at today's #NORDSummit in D.C.

When Mark was born, #hemophilia had no approved treatments. Today, huge progress has been made thanks to Mark's decades of leadership as a #BleedingDisorder internationally. Mark has also championed patient-centered outcomes research, which is critical for rare diseases. Today, he serves on NORD's Board of Directors.

Join us in congratulating Mark on this well-deserved award for his lifetime of dedication to the #RareDisease community!

Learn more at rareimpact.org.
Rare Impact Award 2026 honoree Mark W. Skinner, JD. Lifetime Achievement Award for advancing bleeding disorders advocacy at the state, national, and international levels, championing patient-centered outcomes research, and serving as an advisor for NORD and other organizations.
0
0
6
597
National Organization for Rare Disorders (NORD)
@RareDiseases
Oct 26
During a #NORDSummit panel on moving "Beyond One Disease at a Time: The Future of Individualized Genetic Therapies," Apriligen CEO @Nicholas_Vita had insightful words for #pharma investors:

"What #healthcare investors should be asking is, how do I generate the best risk-adjusted o it anywhere else the way you can in #RareDisease. And that's the argument we should be making with investors, because when you have the regulators giving you tailwinds that have never existed before, when you have the scientific community coming up with innovation like they never have before."

#Investment #GeneTherapy
Quote from NORD Summit speaker Nicholas Vita, CEO of Apriligen: "What health care investors should be asking is, how do I generate the best risk-adjusted return? And you can't do it anywhere else the way you can in rare disease."
0
2
19
2,494
National Organization for Rare Disorders (NORD)
@RareDiseases
Oct 26
We were proud to present a 2026 NORD #RareImpactAward for Youth Leadership to Paridhi Tyagi live at the #NORDSummit in D.C.!

Paridhi founded a #genetics club at her high school through NORD #StudentsForRare, raised awareness in her town on #RareDiseaseDay, and collaborated with kshops where she taught #GirlScouts about inclusion and acceptance of people with #RareDiseases and the science of genetics through DNA extraction and modeling.

Learn more at rareimpact.org.
This image is a promotional graphic for the NORD Rare Impact Award. It features the text "Rare Impact Award" and "Youth Leadership" on a blue background, with a photo of a person smiling. The name "Paridhi Tyagi" is displayed below the photo.
0
3
11
435
National Organization for Rare Disorders (NORD)
@RareDiseases
Oct 26
We were proud and enlightened to hear from our top five #RareDisease research poster presenters on stage at the 2026 #NORDSummit, during a year with a record number of more than 100 posters on display!

Thank you to these presenters from @Merck, @ChildrensNatl, @ParentProjectMD, BDRF.
All five Lightning Round research presenters on stage at the 2026 NORD Summit
1
2
13
569
Load more