@RareDiseases

#NORD has been the voice of the U.S. #RareDisease community for 40+ years strong. Official U.S. sponsor of #RareDiseaseDay. On Bluesky at @ https://xitter.cf/t.co/D7PIT4k0Py

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rarediseases.org
Joined October 2009
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NORD honored @Mighty_Tx with a 2026 Industry Innovator #RareImpactAward for developing the first FDA-approved therapy for #BarthSyndrome. Presented by NORD CEO Pamela Gavin alongside @BarthSyndrome Foundation Co-founder Kate McCurdy, the award celebrates decades of advocacy and research that have brought new hope to families. Learn more: rareimpact.org
NORD President and CEO Pamela Gavin presents the 2026 Industry Innovator Rare Impact Award to Mighty Therapeutics CEO Reenie McCarthy, alongside Barth Syndrome Foundation Co-founder and Board Chair Kate McCurdy.

ALT NORD President and CEO Pamela Gavin presents the 2026 Industry Innovator Rare Impact Award to Mighty Therapeutics CEO Reenie McCarthy, alongside Barth Syndrome Foundation Co-founder and Board Chair Kate McCurdy.

NORD President and CEO Pamela Gavin stands with Mighty Therapeutics CEO Reenie McCarthy and Barth Syndrome Foundation Co-founder and Board Chair Kate McCurdy during the presentation of the 2026 Industry Innovator Rare Impact Award.

ALT NORD President and CEO Pamela Gavin stands with Mighty Therapeutics CEO Reenie McCarthy and Barth Syndrome Foundation Co-founder and Board Chair Kate McCurdy during the presentation of the 2026 Industry Innovator Rare Impact Award.

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NEXT WEEK: How can #RareDisease advocates—patients, caregivers, #healthcare professionals, and others—help shape their state's health policies and programs? Join us on Oct. 22 from 1–2 p.m. ET for our webinar on Rare Disease Advisory Councils and how you can strengthen their work. RSVP: bit.ly/4ADrEIH
A group of people is gathered for a photo at an event organized by NORD and the Rare Action Network. Text on the image reads: "October 22, 2026 | 1PM ET Webinar: Make an Impact with Your State’s Rare Disease Advisory Council. Register Today."

ALT A group of people is gathered for a photo at an event organized by NORD and the Rare Action Network. Text on the image reads: "October 22, 2026 | 1PM ET Webinar: Make an Impact with Your State’s Rare Disease Advisory Council. Register Today."

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▶️ Now on demand: Explore how JAK2 and CALR mutations inform diagnosis, risk assessment, and treatment for #PolycythemiaVera and #EssentialThrombocythemia. This case-driven CME program by @medliveofficial covers treatment sequencing and emerging clinical data. Watch: bit.ly/4hZcghn #MPN #MedEd #Hematology
A medical professional is holding the hand of another individual in a comforting manner. The image promotes an educational program titled "JAK2 and CALR in PV and ET: Precision Management," now available on demand. Logos for "Clinician Education Program," "medlive," and "NORD: National Organization for Rare Disorders" are displayed, along with ".5 CME" in the corner.

ALT A medical professional is holding the hand of another individual in a comforting manner. The image promotes an educational program titled "JAK2 and CALR in PV and ET: Precision Management," now available on demand. Logos for "Clinician Education Program," "medlive," and "NORD: National Organization for Rare Disorders" are displayed, along with ".5 CME" in the corner.

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The National Organization for Rare Disorders (NORD) is working to protect vulnerable populations from losing #Medicaid coverage. If not implemented effectively, new #HR1 community engagement/work requirements could cause eligible people with #RareDiseases to lose their health insurance. NORD has created model language for state Medicaid agencies to incorporate into their programs to help prevent this unintended coverage loss. Read it and share it with your state Medicaid officials: rarediseases.org/medicaid-wo…
The image features a poster from the National Organization for Rare Disorders (NORD). It emphasizes "Access is Non-Negotiable" regarding Medicaid work requirements and model policy language. The highlighted document, “Rare Disease and HR1 Work Requirements: Model Policy Language,” offers adaptable policies to help Medicaid programs prevent coverage loss for people with rare diseases.

ALT The image features a poster from the National Organization for Rare Disorders (NORD). It emphasizes "Access is Non-Negotiable" regarding Medicaid work requirements and model policy language. The highlighted document, “Rare Disease and HR1 Work Requirements: Model Policy Language,” offers adaptable policies to help Medicaid programs prevent coverage loss for people with rare diseases.

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Giving patients a voice in #RareDisease research is important. Making them true partners is the next step. nordsummit.org/ At the 2026 #NORDSummit, President & CEO Pamela Gavin will moderate a panel on how #PatientAdvocacy organizations and industry can make patient partnership a more consistent part of research and drug development. Featuring @OIFoundation, @gbscidp, @TakedaPharma, @BCMHouston & @TexasChildrens, a NORD Rare Disease Center of Excellence. Register to join us Oct. 26–27 in Washington, D.C., or virtually: nordsummit.org/
This image is a promotional poster for the "From Patient Voice to Patient Partnership" event at the NORD Breakthrough Summit, October 25-27 in Washington, D.C. It features five speakers: Lisa Butler from GBS | CIDP International Foundation, Pamela Gavin, MBA from NORD, Tracy Hart from Osteogenesis Imperfecta Foundation, Uthra Sundaram, MBA from Takeda Pharmaceuticals U.S.A. Inc., and V. Reid Sutton, MD from Baylor College of Medicine & Texas Children’s Hospital.

ALT This image is a promotional poster for the "From Patient Voice to Patient Partnership" event at the NORD Breakthrough Summit, October 25-27 in Washington, D.C. It features five speakers: Lisa Butler from GBS | CIDP International Foundation, Pamela Gavin, MBA from NORD, Tracy Hart from Osteogenesis Imperfecta Foundation, Uthra Sundaram, MBA from Takeda Pharmaceuticals U.S.A. Inc., and V. Reid Sutton, MD from Baylor College of Medicine & Texas Children’s Hospital.

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After a life-changing diagnosis of Neuro-Behçet’s disease and years of severe #neurological symptoms, Gary found remission and a new sense of purpose helping others navigate chronic illness and major life transitions. Read his story of resilience and recovery from @VasculitisFound: vasculitisfoundation.org/gar…

Gary Naccarato: Rebuilding a Life After Neuro-Behçet’s Disease

Follow Gary’s journey with Neuro-Behçet’s disease as he rebuilds his life after remission, finding purpose, and hope through chronic illness.

vasculitisfoundation.org
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Today's #FDA announcement is an important step forward for the #raredisease community. Traditional clinical trials aren't always feasible for rare diseases, where patient populations are small and scientific challenges are complex. #NORD welcomes FDA's commitment to developing a new regulatory framework that reflects these realities while maintaining rigorous standards for safety and effectiveness. For millions of patients and families waiting for treatments, getting this right matters. We look forward to working with FDA and the rare disease community to help shape this effort. Read our full statement: rarediseases.org/nord-applau…
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What will it take to accelerate the next decade of #RareDisease innovation? We’re thrilled to have @JazzPharma President & CEO Renee Gala join @CNBC's @BeckyQuick and fellow biopharma leaders at the #NORDSummit for a Rare Disease CEO Leadership Dialogue exploring the decisions and partnerships needed to drive breakthroughs, address the challenge of scale, and advance more treatments for more rare diseases. Join the conversation October 26–27 in Washington, D.C. and virtually. Register at nordsummit.org.
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Join #IndoUSRare and @MitoSociety for the 12th Annual Conference of the Society for Mitochondrial Research and Medicine, convening experts and advocates advancing understanding and care in #MitochondrialDisease. November 19–21, 2026 Narayana Health City, #Bengaluru, India 10 AM–5 PM Be part of #SMRM2026: indousrare.org/smrm/ #mito

Explore 12th SMRM Annual Conference Mitochondrial Research Community

Join IndoUSrare at the 12th Annual SMRM Conference in Bengaluru where SMRM, NIMHANS and IndoUSrare collaborate to advance mitochondrial medicine.

indousrare.org
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Join @MissionMSA for their final Path to a Cure event of 2026! On Sunday, Nov. 8 at 9 a.m. PT, the #MSA community will come together in San Diego, #California to honor and uplift those living with #MultipleSystemAtrophy. Register as an individual, build a fundraising team with loved ones, or make a donation here: missionmsa.org/pathtoacure
Promotional graphic for "Path to a Cure" event in San Diego, California. The event is at De Anza Cove Park on Sunday, November 8, at 9:00am PT. Includes a link to visit missionmsa.org/pathtoacure for more information. Features vibrant road designs and Mission MSA logo.

ALT Promotional graphic for "Path to a Cure" event in San Diego, California. The event is at De Anza Cove Park on Sunday, November 8, at 9:00am PT. Includes a link to visit missionmsa.org/pathtoacure for more information. Features vibrant road designs and Mission MSA logo.

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Cindy Ranii joined Landy Thomas of @wearesrna for an "Ask the Expert: Community Spotlight Edition" podcast, where she talked about her journey with #TransverseMyelitis, or #TM. Listen here: srna.ngo/6ac

Community Spotlight - Cindy Ranii

Community Spotlight - Cindy Ranii | From the SRNA Resource Library for the rare neuroimmune community.

wearesrna.org
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Meet 2026 NORD #RareImpactAwards Community Champion, Julieta Bonvin Sallago, MD! Julieta has been instrumental in engaging #Spanish-speaking communities about #RareDiseases, supporting #Latino patient and family listening sessions, translating NORD Rare Disease Reports, and assisting with recruitment for the Living Rare Study. Join us in celebrating Julieta! Learn more at rareimpact.org.
This image features a banner for the NORD Rare Impact Award. Below the title, there is a portrait of an individual labeled as a Community Champion. The name "Julieta Bonvin Sallago, MD" is displayed at the bottom.

ALT This image features a banner for the NORD Rare Impact Award. Below the title, there is a portrait of an individual labeled as a Community Champion. The name "Julieta Bonvin Sallago, MD" is displayed at the bottom.

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Who better to lead a conversation on the next decade of #RareDisease innovation and impact than @CNBC’s @BeckyQuick? Co-anchor of @SquawkCNBC," host of #CNBCCures, and the parent of a child living with a rare disease, Becky brings a unique perspective to the #NORDSummit. Her extensive experience interviewing some of the world’s most influential business leaders is matched by a deeply personal understanding of the urgent need for more treatments and improved care for people living with #RareDiseases and their families. On October 26, she’ll bring those perspectives together as she moderates a critical conversation with four CEOs on the innovation, investment, challenges, and opportunities that will shape the future of rare disease. Be part of the conversation. Register to join us in Washington, D.C., or virtually today: nordsummit.org
A promotional graphic for the NORD Breakthrough Summit, held October 25-27 in Washington, D.C. It announces "The Next Decade of Rare Disease: A CEO Leadership Dialogue," moderated by Becky Quick, co-anchor of CNBC's "Squawk Box" and host of "CNBC Cures."

ALT A promotional graphic for the NORD Breakthrough Summit, held October 25-27 in Washington, D.C. It announces "The Next Decade of Rare Disease: A CEO Leadership Dialogue," moderated by Becky Quick, co-anchor of CNBC's "Squawk Box" and host of "CNBC Cures."

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Dr. Ayşe Altıntaş joined @wearesrna for an episode of their "Community Meets Clinic" podcast, where she discussed her history in #neuroimmunology, her research interests, the clinic at Koç University School of Medicine, and more. Listen here: srna.ngo/b1guhqx

Community Meets Clinic - Dr. Ayşe Altıntaş

Community Meets Clinic - Dr. Ayşe Altıntaş | From the SRNA Resource Library for the rare neuroimmune community.

wearesrna.org
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How can multidisciplinary care support communication, mobility, and engagement for individuals with #RettSyndrome? For #RettSyndromeAwarenessMonth, explore these approaches in Session 4 of our #Rett education series and earn 0.5 #CME credit today: bit.ly/4yB2ngI
Cover image for a Clinician Education Program featuring information on "Communication, Mobility, Engagement: Multidisciplinary Practices in Rett Syndrome That Transform Outcomes." Logos of Medlive, NORD, the International Rett Syndrome Foundation, and NTOCC are displayed at the bottom. A person is holding a clipboard in the background.

ALT Cover image for a Clinician Education Program featuring information on "Communication, Mobility, Engagement: Multidisciplinary Practices in Rett Syndrome That Transform Outcomes." Logos of Medlive, NORD, the International Rett Syndrome Foundation, and NTOCC are displayed at the bottom. A person is holding a clipboard in the background.

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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 experts across the #CGT field. Register by Oct. 13 to save up to 20%: bit.ly/asgct-breakthroughs
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.

ALT 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.

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How can clinicians better anticipate treatment-related AEs by partnering with caregivers in #RettSyndrome care? For #RettSyndromeAwarenessMonth, explore these strategies in Session 3, Partnering with Caregivers: Anticipating and Managing AEs in Rett Syndrome Treatment. Earn 0.5 #CME credit today: bit.ly/4yB2ngI #RareDisease #MedEd
An individual in a white coat holding a clipboard. The image promotes a clinician education program by medlive, NORD, International Rett Syndrome Foundation, and NTOCC. The topic is "Partnering with Caregivers: Anticipating and Managing AEs in Rett Syndrome Treatment." It is available on-demand and offers .5 CME.

ALT An individual in a white coat holding a clipboard. The image promotes a clinician education program by medlive, NORD, International Rett Syndrome Foundation, and NTOCC. The topic is "Partnering with Caregivers: Anticipating and Managing AEs in Rett Syndrome Treatment." It is available on-demand and offers .5 CME.

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#RareDisease research takes the spotlight at the 2026 #NORDSummit ⚡️ Five researchers, among many others on view, will share emerging findings and new perspectives during Lightning Round Presentations. Join us Oct. 26–27 in Washington, D.C. or virtually: bit.ly/4jPFNwe Feat. #MedicalResearch representatives from @Merck, @EWhiteheadFdn, @APBDRF, @ChildrensNatl & @ParentProjectMD
This image is a poster for the NORD Breakthrough Summit in Washington, D.C., October 25-27. It features headshots and affiliations of six Lightning Round Poster Presenters: Neto Coulibaly from Merck, George Eastwood from the Emily Whitehead Foundation, Lindsay Gill from APBD Research Foundation, Sydney Jones from Children's National Hospital, and Rachel Schrader, a parent project representative for Muscular Dystrophy. The design is set against a blue background with a decorative summit logo.

ALT This image is a poster for the NORD Breakthrough Summit in Washington, D.C., October 25-27. It features headshots and affiliations of six Lightning Round Poster Presenters: Neto Coulibaly from Merck, George Eastwood from the Emily Whitehead Foundation, Lindsay Gill from APBD Research Foundation, Sydney Jones from Children's National Hospital, and Rachel Schrader, a parent project representative for Muscular Dystrophy. The design is set against a blue background with a decorative summit logo.

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Meet 2026 NORD #RareImpactAwards Community Champion, Lily Emmanuel! A passionate organizer in NORD’s #RunningforRare program, Lily helped grow participation out west in her state of #Colorado. She forged partnerships with local organizations and brought the #RareDisease community together to get active and raise critical funds. Learn more at rareimpact.org.
The image features a promotional graphic for NORD's Rare Impact Award. The top displays the NORD logo and the text "Rare Impact Award." Below is a picture of a smiling individual with an orange ribbon stating "Community Champion." The name "Lily Emmanuel" is written at the bottom.

ALT The image features a promotional graphic for NORD's Rare Impact Award. The top displays the NORD logo and the text "Rare Impact Award." Below is a picture of a smiling individual with an orange ribbon stating "Community Champion." The name "Lily Emmanuel" is written at the bottom.

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