National Organization for Rare Disorders (NORD)

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

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

Promotional graphic for the NORD Summit, October 19-21, 2025 in Washington, D.C., featuring a photo of a rare disorder patient speaking at a past event and the headline, "Register by September 8 to save up to $300!"

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

NORD’s 2025 Rare Diseases & Orphan Products Breakthrough Summit to Spotlight CEOs, Investors, and Regulatory Leaders Driving Innovation - National Organization for Rare Disorders “The NORD Breakthrough Summit isn’t just a gathering — it’s a catalyst for solving one of America’s most urgent public health challenges,” said Pamela K.

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

Promotional poster for the NORD Breakthrough Summit from October 19-21, 2025, featuring a speaker at a podium discussing the future of diagnosis and treatment for rare and genetic disorders. Includes a colorful graphic of a city skyline and a call to action to register today.

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

NCSL Legislative Summit in Boston August 4-6, 2025. Find us in the exhibit hall #NCSLsummit.

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-...

Happening today at 2:30pm ET: A vital CME session on #IgAN care, covering mechanisms to management. Proud to partner with @kdigo.org & @nordrare.bsky.social

Don’t miss it 🔗 bit.ly/44ZyEkg

#Nephrology #CKD #MedicalEducation #CME #KDIGO #MedLive

Promotional poster for a NORD webinar titled 'Finding Strength in Self-Acceptance' scheduled for Thursday, July 17 at 6:00 PM EST. Features images and names of three participants: Tahra Anglade, a doctoral student in clinical psychology; Ben Gray, a rare disease community member; and Rae Charlotte, a BSN, RN and community member. The poster has a blue and white color scheme with a logo of NORD at the top

New to your #RareDisease journey or years in, you’re not alone. Join #NORD on July 17 at 6 p.m. ET for “Finding Strength in Self-Acceptance”—a virtual talk on emotional well-being, self-advocacy & connection. RSVP: bit.ly/4nEeTXZ
#RareDiseases #PatientSupport #SupportGroup #SelfAcceptance

Rare Disease Advocacy Opportunity for New York: Apply to Join the NYS Rare Disease Advisory Council. Who can join? Patients, caregivers, advocates, healthcare professionals, scientists, researchers, and insurance representatives.

New Yorkers! Are you passionate about #RareDiseases? Join the #NewYorkState Rare Disease Advisory Council!

We’re looking for individuals from #NewYork with diverse #RareDisease perspectives & experiences to help shape policies that support rare New Yorkers. Apply now: forms.office.com/g/FVwD7VPteQ

Today our #RareDisease community received heartbreaking news. Newly passed legislation will create major barriers to #Medicaid access for millions, including those with #RareDiseases. This puts vital health coverage at risk for those who need it most.

Hear more in #NORD CEO Pam Gavin’s message.

Rare disease action alert! Tell your Representative to REJECT the Senate's Proposed Medicaid Cuts. 1. Call the Capitol Switchboard at 202-224-3121 and ask to be connected to your Representative's office. 2. Take action at the link above (takes 1 minute). Deadline: today and tomorrow.

The #Senate passed legislation that, if signed into law, imposes dramatic and harmful barriers to #Medicaid for #RareDisease patients. Tell your Representative in the House to reject the Senate-passed bill!

1. Call and use this script: bit.ly/4k8Wsro

2. Send this NORD action alert: bit.ly/41uFH3W

Staff from the NORD Rare Disease Center of Excellence at Emory University School of Medicine and Children's Healthcare of Atlanta speaking on stage during a panel discussion at the NORD Living Rare, Living Stronger Georgia event.

NORD Rare Disease Center of Excellence @emorygenetics.bsky.social staff spoke at our #LivingRareGeorgia event!

Dr. Hong Li, a Georgia #RareDisease Advisory Council member, discussed how a #CenterOfExcellence can help you access specialized care.

Find one here: rarediseases.org/center-of-excellence

Dr. Rossana Sanchez (right) with a mother and daughter she treats, posing at the NORD Living Rare, Living Stronger event in Atlanta, Georgia.

#NORD events like today's #LivingRareGeorgia patient and family meeting connect people living with #RareDiseases and doctors who treat them — like #geneticist Dr. Rossana Sanchez of @emorydeptofmed.bsky.social, a NORD Rare Disease Center of Excellence, pictured here (right) with a family she treats!

Tomorrow is #IPKUDay! Check out this great program from the NORD Rare Disease Center of Excellence at @emorygenetics.bsky.social.

NORD offers financial assistance to #PKU patients in need. Apply here: bit.ly/4czUzRE

If you live in #Canada, learn about the Canadian PKU Registry at pkuregistry.ca.

Photo of presenters in the poster hall at the NORD Breakthrough Summit. Abstract submissions due by July 18, 2025 at midnight Eastern time.

🚨 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

Rare Disease Action Alert! New York State: Tell your assemblymember to pass A1296-A today to support a New York Rare Disease Advisory Council.

The #NewYork #RareDisease Advisory Council bill, #A1296A, may be on a debate list in Albany, but here's what's not up for debate: the daily realities of 1.8 million #NewYorkers navigating #RareDiseases. Ask for your Assemblymember's support TODAY: rareaction.org/take-action/#/252

#NYS #NYC #NYleg

If you live in the #Atlanta, #Georgia area, come meet your local #NORD Rare Disease Center of Excellence - the Emory University Human Genetics team, @emorygenetics.bsky.social at @emorydeptofmed.bsky.social and Children's Healthcare of Atlanta - June 27 at the Georgia Aquarium! Visit LivingRare.org.

Attention #sarcoidosis community! Attend the Global Virtual #StopSarcoidosis Summit, June 28-29, a virtual event offering countless helpful resources and 20+ live, interactive virtual sessions with world-renowned medical experts. RSVP here today: loom.ly/aLYtjh0

#FSR2025 #SarcoidosisSummit

We recently attended @nordrare.bsky.social's Rare Disease Scientific Symposium to explore new clinical trial designs, the translation of patient data into insights, & the role of real-world data in shaping education

Thank you to NORD, our partner since 2019, for hosting this event! 🔗 bit.ly/4kwnrhy

Their children have a rare condition. They didn’t know its name – until now. Five families with children who have the newly discovered rare condition, ReNU syndrome, recently gathered in a D.C. park. Scientists believe 100,000 more people have it.

Putting a name to your kid's #RareDisease can be a victory itself. Sometimes, that name doesn't exist.

We're thrilled for the families who helped discover #ReNUSyndrome with experts at @childrensnational.bsky.social, a #NORD Rare Disease Center of Excellence!

www.washingtonpost.com/dc-md-va/202...

Prasad Says Involvement In US FDA Product Approvals Will Mirror Prior CBER Directors The remarks from the new CBER director suggest Prasad will not be involved in most product-specific decisions.

My dispatches from @nordrare.bsky.social's Rare Scientific Symposium:
FDA CBER Director Prasad on his approach to his new role including what seems to be his stump speech re regulatory flexibility.

insights.citeline.com/pink-sheet/r...

Starting Day 2 of the @nordrare.bsky.social Rare Disease Scientific Symposium hearing from Dr. Vinay Prasad, the new director of the Center for Biologics Evaluation and Research. This inaugural conference is occurring at a critical juncture for rare disease innovation and access to treatment.

A round of applause for PARDAC member, Dr. Jerry Vockley! He was just recognized by NORD as a Medical & Scientific Trailblazers His leadership in rare disease research and care is transforming lives across PA and beyond.

cc: #HermanskyPudlakSyndrome #HermanskyPudlak #HPSNetwork‬ @news.upmc.com @pittdeptofmed.bsky.social ‪@argenx.com @bridgebiopharma.bsky.social #UTSW #UTSWMedicine #UPMC #RareActionNetwork #NORD

Promotional poster for NORD Rare Disease Scientific Symposium titled "Advancing Innovation in Rare Disease". The event is scheduled for June 2-3, 2025 in Washington, D.C. The poster features abstract DNA strand graphics with various scientific icons. Text emphasizes "Last Chance to Register".

Final days to register! Join the #NORD Rare Disease Scientific Symposium, June 2-3 in D.C. or virtually. Connect with regulatory and clinical leaders, and strengthen the #RareDisease research ecosystem.

Register now for in-person or virtual: bit.ly/4hV0X7A
Learn more at NORDscience.org