CheckRare @CheckRare
Leading publisher and learning platform focused on rare diseases. Rare Diseases Are Our Focus, Expertise, and Passion. checkrare.com New York Joined December 2016-
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Scott Baver, PhD, Vice President of Medical Affairs at ITF Therapeutics, discusses long-term safety and efficacy data on givinostat for patients with Duchenne muscular dystrophy. checkrare.com/long-term-safe… #CheckRare #DMD #RareGenetic #RareMusculoskeletal #RareNeurology
📢October 7th at 1:00pm EST Join us for a Live CME Webinar on Lysosomal Disorders and the Brain with Ozlem Goker-Alpan, MD, and Raphael Schiffman, MD. Click the link to sign up: us06web.zoom.us/webinar/regist… #CheckRare #LiveCME #RareDisease #LDRTC
Today is #WorldNarcolepsyDay To learn more about narcolepsy and other rare neurological conditions, visit checkrare.com/diseases/neuro… #CheckRare #Narcolepsy #RareNeurology
Neuroblastoma is a rare childhood cancer, but it is the most common extracranial solid tumor in children. Learn more on our Learning Page at checkrare.com/neuroblastoma/ #CheckRare #Neuroblastoma #RareCancer
Dayne Voelker, MD, discusses a newly developed tool, called the Mastocytosis Control Test (MCT). checkrare.com/a-new-test-for… #CheckRare #RareDisease #Mastocytosis
CheckRare is proud to be covering #ANA2025. Stay tuned for new Neurological content.
Today is #WorldLymphomaAwarenessDay Learn more about the many rare types of this cancer at checkrare.com/diseases/cance… #CheckRare #RareCancer
Unlock the Future of Rare Disease Trials at the 2nd annual Patient Recruitment for Rare Disease Trials Summit on November 4-6, 2025 in Boston, MA. ter.li/staty4
On the final day of Rare Trials Summit, Wendi Erler from Sarepta, Karin Holzer from BIO, and Mandi Waters from Sciensus lead an interactive session “Real-World Evidence that Matters: A Hands-On Workshop in Building Credible Value Stories” #RareTrialsSummit
September is Histiocytosis Awareness Month! Histiocytosis is a rare hematologic disorder characterized by the overproduction of histiocytes, leading to granulomas in many areas of the body. In about half of the cases, genetic changes in the BRAF gene appear to be involved;…
CheckRare is proud to be a partner with the Rare Trials Summit 2025. We attended yesterday afternoon’s Fireside Chat: Beyond Biotech: The Rise of Nontraditional Sponsors on Rare Disease Trials featuring Craig Martin, Ilan Ganot, Casey McPherson, and Ethan Perlstein.
A survey of adults and caregivers of children with X-linked hypophosphatemia (XLH) revealed a broad impact on patients’ lived experiences, including out-of-pocket expenses; physical, mental, and social health, and challenges accessing expert care. Learn more:…
CME: Myasthenia Gravis Clinical Research Highlights: AAN 2025 Learn more at checkrare.com/learning/p-mya… #CheckRare #CME #RareAutoimmune #RareNeurology #RareMusculoskeletal #MyastheniaGravis
While clinical judgment is irreplaceable, new CTCL staging tool can provide clinicians information on the often complicated staging process, supporting more consistent and informed care. Visit PROBEinCTCL.com for information and resources about CTCL.…
📢September 11th at 10:00am EST Join us for a Live CME Webinar on Case Studies in Diagnosing and Managing FOP with Christiaan Scott, MD, and Raphaella Stander, MD. Click the link to sign up: us02web.zoom.us/webinar/regist… #CheckRare #LiveCME #RareDisease #FOP
Clinical research leaders in HAE provide an overview of the discrepancies in care observed in certain patient populations, as well as a discussion on best practices to reduce those inequalities moving forward. checkrare.com/improving-heal… #CheckRare #RareDisease #RareGenetic
CME: Fabry Disease Research Highlights Learn more at checkrare.com/learning/p-fab… #CheckRare #CME #FabryDisease
📢CME Program: Jean Elwing, MD, of the University of Cincinnati College of Medicine provides an overview of the latest clinical research about PAH presented at CHEST 2024. checkrare.com/learning/p-pah… #CheckRare #RareDisease #RareLung #CMEProgram
CTCL is a rare blood cancer that presents unique challenges in its diagnosis. A new interactive tool is aiming to provide physicians information on the complicated staging of patients with mycosis fungoides or Sézary syndrome, two subtypes of CTCL. Visit PROBEinCTCL.com…
Consider Rare: Suspecting and Diagnosing Fibrodysplasia Ossificans Progressiva (FOP) Ellen Elias, MD and Christiaan Scott, MD, examine best practices to suspect and diagnose this ultra-rare condition. checkrare.com/suspecting-and… #CheckRare #ConsiderRare #FOP

Effie Parks @OnceUponAGene
6K Followers 4K Following Rare Disease Advocate | Award Winning Podcaster | Speaker | Captain Connection | RareMama to my sweet, Ford, who lives with #CTNNB1 🦓
RARE Revolution Magaz... @RareRevolutionM
12K Followers 7K Following Digital magazine giving a voice to those affected by rare conditions and the charities that support them. Contact us: [email protected]
Beacon for Rare Disea... @RareBeacon
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Global Genes @GlobalGenes
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Stephanie Fischer @RarePOV
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KIF1A.ORG @KIF1A
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AllStripes @_allstripes
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Alok Tayi @aloktayi
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n-Lorem Foundation @n_lorem
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heidi bjornson @heidibp
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Cambridge Rare Diseas... @camraredisease
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dazzle4rare @dazzle4rare
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Muscular Dystrophy As... @MDAorg
21K Followers 2K Following MDA is the #1 voluntary health organization in the United States for people living with #MuscularDystrophy, #ALS, and related #neuromuscular diseases.
Marni Cartelli @Purrfectly_Rare
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SYNGAP1 Foundation @Syngap1Fnd
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Bernadette Sheehan Gi... @sheehangilroy
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NewMexicoRareDisease @NMRareDisease
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conical pharmaceutica... @conicalphama
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subramaniyan k @KSubramani15
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RivaPoe @1o1Vd8C71Xnwkdm
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Dr. Debster✌️ @RGVTuffy
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BlueSkyReport @BlueSky_Report
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AI_Portfolio🇺🇸 @Qeeealir695
30 Followers 2K Following 15-30% Monthly | 2 High-Conviction Stocks.Short-Term Gains: 15-20% in Days/Weeks.DM "JOIN" for WhatsApp Alerts. Live Trade Signals • Market Analysis
ARENSIA Exploratory M... @ARENSIA_Clinics
80 Followers 347 Following A German operator of proprietary Research Clinics, dedicated to performing early phase clinical trials involving patients across numerous therapeutic areas.
Rodrigo Hernández Po... @Rodher1990
133 Followers 886 Following
Jacks Miracle Mission @JacksMission
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Metabolic Support UK @weareMSUK
397 Followers 407 Following Metabolic Support UK are the leading patient organisation for Inherited Metabolic Disorders supporting thousands of patients worldwide.
Sleeping Angel Treasu... @sa_treasures
55 Followers 144 Following Author & Graphic Designer. I share Quotes, Books & Life. Mom to 3 boys, 2 in heaven, 1 on earth. ✨ Bringing joy with fun tees, cozy mugs, & more!
Friends of the PBC Fo... @FriendsofPBC
54 Followers 165 Following Our mission is to improve the lives of the 130,000+ 🇺🇸 living with Primary Biliary Cholangitis (#PBC) through comprehensive support, education & advocacy
Christy @SusanBa53774289
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Avwobfu @Avwobfu65563
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Burcu Öztürk Hişmi @BurcuHismi
416 Followers 1K Following Çocuk Metabolizma Hastalıkları Uzmanı • Inherited Metabolic Diseases • Doçent Doktor @MarmaraTıp AAAL96 AnkaraTıp02
Hunter @Hunter140033
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Howard0918 @Howard820918
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Sowbhagya B A @sowbhagya_a
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Alford Beahan @ABeahan58208
100 Followers 4K Following
L Brielmaier @BrielmaierL
543 Followers 479 Following Engineer, @IEEEBrain, IEEE EMB, PLWALS, prog-rock, “Scientists investigate that which already is. Engineers create that which has never been.” Albert Einstein
Christiana Madonna @InspireMedia6
2K Followers 7K Following Life has got all those twists and turns. You've got to hold on tight and off you go
Eric Odom @odome5
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Natasha Mayer @NatashaMay73215
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DayTradeAlerts🇺�... @Soupi132636
52 Followers 2K Following 15-30% Monthly | 2 High-Conviction Stocks.Short-Term Gains: 15-20% in Days/Weeks.DM "JOIN" for WhatsApp Alerts. Live Trade Signals • Market Analysis
iPain Foundation Rese... @iPain_Research
3K Followers 3K Following Supporting @iPainOfficial & @powerofpain Research Projects #NERVEmber
CureDuchenne @CureDuchenne
6K Followers 588 Following We are committed to improving the lives of those affected by Duchenne through accelerating research, improving care and empowering the community.
Octavia Beatty-Maggio @BeattyMagg1425
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Karan Srisurapanont, ... @KSrisurapanont
54 Followers 433 Following MD from Chiang Mai University 🇹🇭| DV2025 winner 🇺🇸 | Aspiring internist | Hematologic malignancies | AAMC ID 16254545 | #IMG | #Match2026
Andy @imandyevans
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Francisco Ureta @FranciscoUreta4
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Laliekar @Laliekar774013
129 Followers 3K Following
Marian Viñas @Marianvinas
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Effie Parks @OnceUponAGene
6K Followers 4K Following Rare Disease Advocate | Award Winning Podcaster | Speaker | Captain Connection | RareMama to my sweet, Ford, who lives with #CTNNB1 🦓
National Organization... @RareDiseases
40K Followers 3K Following #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://t.co/D7PIT4k0Py
RARE Revolution Magaz... @RareRevolutionM
12K Followers 7K Following Digital magazine giving a voice to those affected by rare conditions and the charities that support them. Contact us: [email protected]
Beacon for Rare Disea... @RareBeacon
12K Followers 10K Following Beacon is a UK-based charity that is building a united rare disease community with patient groups at its heart. Previously known as Findacure.
Rare Disease Day @rarediseaseday
42K Followers 3K Following 29 February 2024 is Rare Disease Day. Raising awareness for patients, families and carers around the world that are affected by rare diseases. #RareDiseaseDay
Patient Worthy @PatientWorthy
8K Followers 7K Following We're a resource for engaging, informative content and rare patient news, well done.
Luke Rosen @lukebrosen
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Global Genes @GlobalGenes
29K Followers 6K Following Empowering the Next Generation Rare Disease Advocate. Merged with RARE-X Dec. 2022. #CareAboutRare
EveryLife Foundation @EveryLifeOrg
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EURORDIS-Rare Disease... @eurordis
32K Followers 1K Following An alliance of over 1,000 patient organisations working across borders and diseases to improve the lives of all people living with rare diseases.
Stephanie Fischer @RarePOV
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2K Followers 3K Following We help clients find rare & non-rare disease patients & caregivers for research studies, & connect patients & caregivers with paid research opportunities.
KIF1A.ORG @KIF1A
1K Followers 589 Following We connect families affected by KIF1A and relentlessly work to accelerate research. We need to find treatment for this rare neurodegenerative disease. Fast.
AllStripes @_allstripes
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Alok Tayi @aloktayi
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David Fajgenbaum, MD @DavidFajgenbaum
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RarasNoInvisibles @NoInvisibles
55K Followers 7K Following Hablamos de salud, enfermedades raras, inclusion social y biomedicina. Escribe @Sombradoble Mas Información: [email protected]
heidi bjornson @heidibp
723 Followers 1K Following Rare Disease Advocate, #PCD, #RareAsOne Program Manager, @CZIScience, Attorney, Mom
Adam Feuerstein ✡�... @adamfeuerstein
121K Followers 780 Following Biotech reporter @statnews. Dog ❤️er. Polk Award winner. #COYS. Said one analyst: The likes of Adam Feuerstein attack viciously. On Signal: stataf.54
Genetic Alliance UK @GeneticAll_UK
15K Followers 2K Following National charity working for everyone affected by genetic, rare and undiagnosed conditions. We run the campaign Rare Disease UK and support network @SWAN_UK.
National Bleeding Dis... @nbd_foundation
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L Brielmaier @BrielmaierL
543 Followers 479 Following Engineer, @IEEEBrain, IEEE EMB, PLWALS, prog-rock, “Scientists investigate that which already is. Engineers create that which has never been.” Albert Einstein
National Psoriasis Fo... @NPF
23K Followers 858 Following The mission of the National Psoriasis Foundation is to drive efforts to cure psoriatic disease and improve the lives of those affected.
Allyson M. W. Dyar @Allyson13
797 Followers 248 Following Tweets since 2008--Boomer Geekette & Star Trek Fan! Proud Cat Mom 😸Book Reviewer 📚(science) for @NetGalley. alumna Iona U, Troy U🧵🦋AllysonThirteen
Washington University... @WashURareCare
67 Followers 69 Following NORD Rare Disease Center of Excellence | We provide clinical diagnosis, care for those with rare diseases and are recognized as a leader in rare diseases.
UCDavis_Gene_Therapy @UCDavisGT
2K Followers 2K Following Gene Therapy Center at UC Davis Health in Sacramento. We currently have 59 clinical trials of gene and/or cell therapy at UCD & a GMP Facility. Keeping busy! 😊
Leslie Karras @LKarras
254 Followers 1K Following Experienced Regional Sales Manager, sales professional who possesses a strong work ethic, and a deep understanding of the medical sales industry.
Stronger Than Autoimm... @strngr_autoimun
52 Followers 109 Following Corporate Health Advocate🩺 Autoimmune Coach 💪 Helping patients + companies navigate autoimmune health DM for coaching & consulting
Linda Molina @LindaMolina_
246 Followers 2K Following I am a #medfrag mom. Bringing awareness to and advocating for the needs of medically fragile children and their families.#RareDisease #COXPD4 #mito
StephyDinMD @StephDinDC
717 Followers 543 Following Comedy🎙Sports ⚽️ Music 🎵 Tweets are mine & mine only.
Protagonist Therapeut... @ProtagonistTx
352 Followers 211 Following Discovering and developing novel therapies through our proprietary peptide technology platform. #biotech #lifesciences #innovation
Malay B. Shah @TxpDoc
978 Followers 1K Following Liver Transplant Surgeon, Defender of the Downtrodden, #TransplantTwitter, #MedTwitter, #LiverTwitter, tweets and retweets are my own.
Jay Gordon, MD, FAAP @JayGordonMDFAAP
18K Followers 4K Following Pediatrician/Activist/Soccer Player/Lapsed Surfer/Self-proclaimed clinical COVID expert
Dyne Therapeutics @Dyne_tx
1K Followers 317 Following A clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases: https://t.co/0XslT2HWqg
Deciphera Pharma @Deciphera
860 Followers 169 Following Deciphera is a biopharmaceutical company focused on discovering, developing & commercializing important new medicines to improve the lives of people with cancer
Day One Biopharmaceut... @DayOneBio
1K Followers 222 Following We are a new biotech company focused on developing new cancer therapies for patients of all ages.
Daiichi Sankyo US @DaiichiSankyoUS
4K Followers 386 Following We create life-changing solutions for patients. See our guidelines: https://t.co/A1iZajC0s5
Bionews @bionewsservices
574 Followers 9 Following Bionews is a digital health solutions company that empowers rare disease communities with trusted information & news, fostering a space where hope thrives.
Cytokinetics @Cytokinetics
2K Followers 748 Following We are developing potential medicines to improve the healthspan of people with diseases of impaired muscle function. 2025 Great Place to Work-Certified🏅
Gilead Sciences @GileadSciences
73K Followers 197 Following At Gilead, we set – and achieve – bold ambitions to create a healthier world for all people. Read more: https://t.co/TZWHS6iRTe
Cyclo Therapeutics, I... @Cyclodexpert
681 Followers 143 Following Dedicated to developing life-changing medicines for patients and families living with challenging diseases
Cycle Pharmaceuticals @CyclePharma
296 Followers 192 Following Passionate about providing quality treatments & services. Everything we do is with patients & caregivers’ best interests at heart. Every single patient matters.
CSL @CSL
6K Followers 234 Following Since our start in 1916, we have been driven by our promise to identify, develop and deliver innovations so our patients can live life to the fullest.
CRISPR Therapeutics @CRISPRTX
23K Followers 0 Following
Congenica @Congenica
2K Followers 533 Following We are Congenica, driving #PrecisionMedicine by providing automated software and solutions for the analysis and interpretation of genomic data at scale.
ERN ReCONNET 🇪🇺 @ern_reconnet
3K Followers 883 Following 🔬🩺 European Reference Network on Rare and Complex Connective Tissue and Musculoskeletal Diseases #ERNReCONNET
ERN eUROGEN @ERN_eUROGEN
1K Followers 536 Following Improving diagnosis, treatment & care for rare uro-recto-genital diseases & complex conditions needing highly specialised surgery. Funded by the EU.
Cogent Biosciences @CogentBio
2K Followers 28 Following Precision therapeutics for genetically defined diseases
Clene Nanomedicine @CleneNano
840 Followers 170 Following Clene is committed to revolutionizing the treatment for people living with neurodegenerative diseases to restore and protect neuronal health.
Centene @Centene
6K Followers 74 Following Transforming the health of the communities we serve, one person at a time.
Cellectar Biosciences @CellectarBio
813 Followers 1K Following Learn more about the pediatric HGG clinical study at https://t.co/N9QWzbiN3k
Celldex Therapeutics @Celldex
179 Followers 65 Following We are dedicated to developing monoclonal and bispecific antibodies that address devastating diseases for which available treatments are inadequate.
Castle Creek Bioscien... @CastleCreekBio
462 Followers 94 Following Dedicated to making life better for people living with genetic diseases.
Cartesian Therapeutic... @CartesianTX
949 Followers 73 Following Clinical-stage company pioneering mRNA cell therapies for the treatment of autoimmune diseases.
Caribou Biosciences @CaribouBio
7K Followers 209 Following Clinical-stage biopharmaceutical company developing transformative therapies to treat devastating human diseases
Capricor Therapeutics @Capricor
2K Followers 241 Following Capricor (NASDAQ: $CAPR) is a biotech company focused on the development of cell & exosome-based therapeutics for treatment and prevention of serious diseases.
Cabaletta Bio @CabalettaBio
511 Followers 151 Following Transforming the treatment of B cell-mediated autoimmune diseases
BrainStorm @BrainstormCell
5K Followers 772 Following BrainStorm Cell Therapeutics, Inc. (NASDAQ: BCLI) is a clinical-stage biotechnology company developing autologous cell therapies for neurodegenerative diseases.
Genetix Biotherapeuti... @genetix_biotx
10K Followers 724 Following We’re transforming lives through curative #genetictherapies - building a future of lasting hope for patients and families. https://t.co/1dUPTEWcyx
Biophytis @biophytis
2K Followers 119 Following We are a clinical-stage biotechnology company focused on the development of drug candidates for the treatment of age-related diseases #Sarcopenia #DMD
BioMarin @BioMarin
742 Followers 0 Following At BioMarin, we are committed to transforming lives through genetic discovery. Our community guidelines: https://t.co/w1kvdgjTYG
BioLineRx Limited @BioLineRx_Ltd
427 Followers 264 Following About BioLineRx BioLineRx Ltd. is a pre-commercial stage biopharmaceutical company pursuing life-changing therapies for certain cancers and rare diseases.
BioCryst Pharmaceutic... @BioCrystPharma
2K Followers 51 Following We develop oral small-molecule & protein therapeutics for hereditary angioedema (HAE) & other rare diseases. Community guidelines: https://t.co/HJsf9ax79K.
BeyondSpring @BeyondSpringInc
453 Followers 8 Following BeyondSpring Pharmaceuticals is a global biopharmaceutical company focused on developing innovative cancer therapies to improve clinical outcomes.
CG Life @cglifeagency
2K Followers 3K Following Strategic #MarketingAgency shaped by science. Our science-centered creativity helps tell your story. Let’s shape the future together. #LifeScienceMarketing
BeiGeneGlobal @BeiGeneGlobal
15 Followers 2 Following BeOne Medicines USA, Inc. is the U.S. affiliate of BeiGene, Ltd., a global oncology company. Visit https://t.co/9mTRBkYuRg for the latest information.
Beam Therapeutics @BeamTx
5K Followers 202 Following Our vision is to provide life-long cures for patients suffering from serious diseases. #DaretoBeam #BeamTeam
Baxter International @baxter_intl
16K Followers 6 Following A global medtech leader redefining how healthcare is delivered to make a greater impact today, tomorrow & beyond. This content is intended for a global audience
BachPharma @BachPharma
12 Followers 37 Following Help Us, Help Millions. Change the way we treat neurodegenerative disease. Learn about Bach Pharma's revolutionary new approach at https://t.co/cSU2yCa5ST