Rare Diseases @CheckOrphan
CheckOrphan is the leading #news and information platform dedicated to #rarediseases and #orphandrugs checkorphan.org Santa Cruz, USA / Basel, CH Joined April 2009-
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Climb Bio Announces CLYM116 Preclinical Data Highlighting Potential for Best-In-Class Therapeutic for IgAN - For More Information Visit shorturl.at/LkaPs @ClimbBio #IgAN #IgA_Nephropathy #Rare_Diseases
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Dyne Therapeutics Receives Orphan Drug Designation in Japan for DYNE-251 in Duchenne Muscular Dystrophy - For More Information Visit shorturl.at/Jn05L @Dyne_tx #Duchenne #Muscular_Dystrophy #Rare_Diseases
Acoramidis Begins to Reduce Cumulative Cardiovascular Outcomes Within the First Month of Treatment in Patients with ATTR-CM - For More Information Visit shorturl.at/8mxc5 @BridgeBioPharma #ATTR_CM #Transthyretin_Amyloid_Cardiomyopathy #Rare_Diseases #OrphanDrugs
New Givinostat Data in Duchenne Muscular Dystrophy Presented at NMSG 2025 - For More Information Visit shorturl.at/QwnGx #Italfarmaco #Duchenne #DMD #Rare_Diseases #OrphanDrugs #Muscular_Dystrophy
Savara Presents New Data From the Phase 3 IMPALA-2 Clinical Trial of Molgramostim in Patients With Autoimmune Pulmonary Alveolar Proteinosis (aPAP) - For More Information Visit shorturl.at/LLk7j @SavaraPharma #aPAP #Pulmonary_Alveolar_Proteinosis #Rare_Diseases
ANGLE Unveils Breakthrough Data Demonstrating Potential for Minimally Invasive Monitoring of Glioblastoma Through Circulating Tumour Cells - For More Information Visit shorturl.at/uJs4x #Brain_Cancer #GBM #Glioblastoma #Rare_Diseases #OrphanDrugs #ANGLE
Pooled Data Presented at ERS: Nerandomilast Monotherapy Linked to Nominally Significant Reduction in Risk of Death in IPF And PPF - For More Information Visit shorturl.at/IugNl @Boehringer #Rare_Diseases #OrphanDrugs #IPF #PPF #Idiopathic_pulmonary_fibrosis
Larimar Therapeutics Announces Positive Data from Ongoing Long-term Open Label Study and Updates to Nomlabofusp Program for Friedreich’s Ataxia - For More Information Visit shorturl.at/cnPKM @larimarthrx #Friedreich_Ataxia #Rare_Diseases #OrphanDrugs #FA
Novo Nordisk Submits Biologics License Application (BLA) to FDA for Mim8; An Investigational, Prophylaxis Treatment for People with Hemophilia A with or without Inhibitors - For More Information Visit shorturl.at/1htTu @novonordisk #Hemophilia #Rare_diseases
Tonix Pharmaceuticals Plans to Initiate Prader-Willi Syndrome Phase 2 Trial of TNX-2900 (Intranasal Potentiated Oxytocin) in 2026 - For More Information Visit shorturl.at/tn6fb @TonixPharma #Rare_Diseases #PWS #Prader_Willi_Syndrome #OrphanDrugs #Drug_Research
PureTech’s Deupirfenidone Shows Lung Function Stabilization in IPF Patients Switching from Placebo or Pirfenidone in Phase 2b ELEVATE Trial Extension - For More Information Visit shorturl.at/u1ujS @PureTechH #IPF #Rare_Diseases #idiopathic_pulmonary_fibrosis
Immuneering Announces Extraordinary 86% Overall Survival at 9 Months in First-Line Pancreatic Cancer Patients Treated with Atebimetinib + mGnP - For More Information Visit shorturl.at/YQRBE #Rare_diseases #Pancreatic_Cancer @Immuneering #OrphanDrugs #Cancer
Crinetics Announces FDA Approval of PALSONIFY™ (Paltusotine) for the Treatment of Adults with Acromegaly - For More Information Visit shorturl.at/ivKBg @Crinetics #Acromegaly #Rare_Diseases #FDA #OrphanDrugs #Medical_Research #Crinetics #Drug_Research
Kura Oncology and Kyowa Kirin Announce Publication of Pivotal Ziftomenib Data in R/R NPM1-Mutated Acute Myeloid Leukemia in the Journal of Clinical Oncology - For More Information Visit shorturl.at/aUNZM @kuraoncology #AML #Leukemia #Rare_Diseases #OrphanDrugs
Arialys Therapeutics Receives FDA Rare Pediatric Disease Designation for ART5803 to Treat Anti-NMDA Receptor Encephalitis (ANRE) - For More Information Visit shorturl.at/neXyJ @ArialysRx #Rare_Diseases #ANRE #OrphanDrugs #Encephalitis #Drug_research #FDA
FDA Granted Orphan Drug Designation to Biostar Pharma’s Utidelone for the Treatment of Pancreatic Cancer - For More Information Visit shorturl.at/O3k7h #Biostar_Pharma #Pancreatic_Cancer #Rare_Diseases
Clene Presents New Clinical Data at ECTRIMS 2025 Demonstrating CNM-Au8® Improves Brain Energy Metabolism in Multiple Sclerosis Patients - For More Information Visit shorturl.at/SrdEa @CleneNano #MultipleSclerosis #OrphanDrugs #Rare_Diseases #Drug_research
Evkeeza® (evinacumab-dgnb) ANGPTL3 Antibody Approved in the U.S. for Children as Young as 1 Year Old with Homozygous Familial Hypercholesterolemia (HoFH) - For More Information Visit shorturl.at/H3E2a #HoFH #Rare_Diseases #OrphanDrugs @Regeneron #Drug_research
CSL Vifor and Travere Therapeutics Highlight Inclusion of FILSPARI® in Updated 2025 KDIGO Guidelines for IgA Nephropathy Treatment - For More Information Visit shorturl.at/26zju @CSL @TravereRare #Rare_Diseases #OrphanDrugs #IgANephropathy #IgAN #Drug_research
MAIA Biotechnology Awarded $2.3 Million Grant by the NIH for THIO-101 Phase 2 Trial in Advanced Non-Small Cell Lung Cancer (NSCLC) - For More Information Visit shorturl.at/YnXLW @MAIABiotech #Rare_Diseases #NSCLC #Lung_Cancer #OrphanDrugs #NIH #Drug_research
Rare Disease Day @rarediseaseday
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Maria Teresa Piedra P... @MTPP5001
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Shawn Friesen @ShawnRFriesen
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My Swagz @my_swagz
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𝕊 Bitcoin 𝕊 @Charts4bitcoin
420 Followers 2K Following Interests: #GROK journalism, #AI assistants, Trading #Algorithms (Investor in #Bitcoin and #Ethereum), and lots in between (Enjoy)
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
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.
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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Effie Parks @OnceUponAGene
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Raras pero Reales @RarasReales
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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
FEDER | Enfermedades ... @FEDER_ONG
39K Followers 8K Following 👉🏻 Somos la esperanza de 3 millones de personas con #enfermedadesraras 🍀 Representamos a 442 organizaciones de pacientes y a 1.546 patologías poco frecuentes
Cambridge Rare Diseas... @camraredisease
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Patient Worthy @PatientWorthy
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Ethan Perlstein bio/a... @eperlste
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55K Followers 7K Following Hablamos de salud, enfermedades raras, inclusion social y biomedicina. Escribe @Sombradoble Mas Información: [email protected]
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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.
ERDERA @ERDERA_org
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Genetic Alliance UK @GeneticAll_UK
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Rare Disease UK @rarediseaseuk
21K Followers 2K Following National campaign run by @GeneticAll_UK to improve the lives of those affected by rare conditions and all who support them. #RareDisease.
Luke Rosen @lukebrosen
2K Followers 423 Following Dad of two remarkable kids. Founder of https://t.co/GkrwXhfkoy & https://t.co/eWtcezWeJc. Works with families affected by neurological diseases & cancer. Firefighter. Baseball & hockey.
Climb Bio @ClimbBio
71 Followers 56 Following We believe elevating relationships leads to more meaningful insights, better answers, and ultimately, to more inspired medicines for patients
BridgeBio Pharma @BridgeBioPharma
2K Followers 165 Following Hope Through Rigorous Science Our mission is to discover, create, test and deliver transformative medicines to treat patients with genetic diseases. $BBIO
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29 Followers 5 Following 🌟 Discover the essence of creativity with Larimar TX. Designer, artist, and visionary bringing unique designs and innovative ideas to life. 🌟
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Aro Biotherapeutics @AroBioTx
127 Followers 52 Following Unlocking the Potential of Genetic Medicines
Monopar Therapeutics @MonoparTx
153 Followers 227 Following Clinical-stage biopharma focused on developing therapeutics designed to extend life or improve the quality of life of cancer patients
Palvella Therapeutics @PalvellaTX
585 Followers 105 Following Palvella Therapeutics ($PVLA) is #biotech focused on novel therapies for patients with serious, rare genetic skin diseases w/ no FDA approved therapies.
中外製薬 @chugai_cc
8K Followers 23 Following 「創造で、想像を超える。」中外製薬の公式アカウントです。当社の企業情報や採用情報、社会貢献活動、疾患啓発等に関する情報を発信します。 ソーシャルメディアポリシー：https://t.co/m6GBIofXSS
StealthBT @StealthBT
1K Followers 125 Following We’re driven by a desire to help those with mitochondrial dysfunction, leading mitochondrial medicine for both rare and common diseases. https://t.co/45rgM9EXLN
Syndax @Syndax
565 Followers 156 Following Syndax is a commercial-stage biopharmaceutical company advancing innovative cancer therapies. Guidelines: https://t.co/Fjrc9AdChk
Skyhawk Therapeutics @Skyhawk_Tx
672 Followers 204 Following Skyhawk Therapeutics is a drug discovery and development company focused on revolutionizing disease treatment with small molecules that modify RNA expression.
Aptevo Therapeutics @APVO98121
127 Followers 142 Following Developing safe, effective bispecific therapeutics for cancer patients with blood and solid tumor malignancies.
Novocure @Novocure
3K Followers 230 Following We strive to extend survival in some of the most aggressive forms of cancer. Patient images reflect patient(s) health status when photographed.
Acadia Pharmaceutical... @AcadiaPharma
1K Followers 109 Following Acadia is trailblazing breakthroughs in science to elevate life. See our community guidelines: https://t.co/1v9MHwVi9s
UCL @ucl
141K Followers 897 Following Sharing highlights of life at UCL (University College London), London’s leading multidisciplinary university. Follow @uclnews and @uclevents.
GRI Bio, Inc. @GRI_BIO
3K Followers 46 Following GRI Bio is advancing an innovative pipeline of Natural Killer T cell modulators for the treatment of inflammatory, fibrotic and autoimmune diseases. $GRI
Amneal Pharmaceutical... @AmnealPharma
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LinusBio @linusbio
102 Followers 12 Following We aspire to transform the way complex health conditions are diagnosed and treated. #Autism #ASD #ALS
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343 Followers 122 Following Our mission is to improve the lives of patients with cancer, their families, and their caregivers.
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RyvuTx @RyvuTx
761 Followers 11 Following Ryvu Therapeutics is developing novel small molecule therapies that address clinical limitations of current treatments in oncology.
NeuroNOS @Neuro_NOS
101 Followers 460 Following A Biotech company solely focused on brain disorders, pioneering a unique platform to develop a new class of therapies for Autism, Alzheimer's, and Glioblastoma.
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Mitsubishi Tanabe Pha... @MTPA_US
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Andelyn Biosciences @AndelynBio
122 Followers 54 Following Andelyn Biosciences is a biopharmaceutical CDMO Pioneering Solutions That Turn Hope into Reality™
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Immunovant @immunovant
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C4 Therapeutics @C4Therapeutics
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United Therapeutics M... @UTHRMed
402 Followers 135 Following Dedicated to providing the latest science & UT innovation to HCPs. See our community guidelines: https://t.co/dLy9hFUwjV
INOVIO Pharmaceutical... @InovioPharma
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422 Followers 75 Following BioVersys AG is a privately owned Swiss pharmaceutical company focusing on R&D of small molecules acting on novel bacterial targets.
Gamida Cell @GamidaCellTx
845 Followers 147 Following Working to turn cells into powerful therapeutics for patients with cancer.
Alkermes @Alkermes
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354 Followers 1 Following Bringing a new era of GPCR medicines to patients with its Native Complex™ platform.
ReAlta Life Sciences @ReAltaLS
27 Followers 34 Following Dedicated to harnessing the power of the immune system to address life threatening acute inflammatory and rare diseases.
Tivic @TivicOfficial
515 Followers 2K Following News in emerging #neuromodulation & #innovation #medicaldevices. Transforming #sinus #allergy solutions without medication: ClearUP Sinus Pain Relief device.
PureTech @PureTechH
4K Followers 1K Following We're a hub-and-spoke biotherapeutics company dedicated to giving life to science and transforming innovation into value for patients and shareholders
Rein Therapeutics @Rein_Tx
25 Followers 45 Following Rein Therapeutics is advancing a novel pipeline of first-in-class medicines to address unmet medical needs in orphan pulmonary and fibrosis indications.
Rocket Pharmaceutical... @RocketPharma
1K Followers 208 Following Seeking #GeneTherapy cures for patients with rare and devastating diseases.
Precigen @Precigen
905 Followers 167 Following Precigen (Nasdaq: PGEN) is a biopharmaceutical company specializing in the advancement of innovative precision medicines to address difficult-to-treat diseases
Scholar Rock @ScholarRock
671 Followers 108 Following Biopharma leader in the biology of the #TGFbeta superfamily of protein growth factors. Community Guidelines: https://t.co/VU9mLWhf59
Cue Biopharma @CueBiopharma
589 Followers 463 Following Developing a novel class of therapeutic biologics to selectively engage and modulate disease-relevant T cells for the treatment of autoimmune disease and cancer
Insmed @Insmed
2K Followers 77 Following We're a global biopharmaceutical company striving to transform the lives of patients facing serious diseases. Community guidelines: https://t.co/Mxp3SAUmXs
Neurotech Pharmaceuti... @NeurotechPharma
68 Followers 42 Following Neurotech Pharmaceuticals, Inc. is a private biotech company focused on developing transformative therapies for chronic eye diseases.
TizianaLifeSciences @TizianaLS
2K Followers 381 Following Developing the worlds only fully human Anti-CD3, foralumab delivered nasally for treatment of Secondary Progressive Multiple Sclerosis, Alzheimer's & ALS. $TLSA
Stoke Therapeutics @StokeTx
2K Followers 173 Following Stoke Therapeutics is addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines.
Osteosarcoma Now @OsteosarcomaNow
346 Followers 209 Following Information and resources for people affected by osteosarcoma. Managed by @BardoFoundation #RareCancer #Osteosarcoma #childhoodcancer
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