Rare Diseases
@CheckOrphan
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CheckOrphan is the leading #news and information platform dedicated to #rarediseases and #orphandrugs
Santa Cruz, USA / Basel, CH
Joined April 2009
Kyowa Kirin and Orchard Therapeutics Announce MHLW Grants Orphan Regenerative Medicine Designation to OTL-200 Gene Therapy for Metachromatic Leukodystrophy - For More Infos Visit https://t.co/9tGqQFLEBP
#Leukodystrophy #MLD #Kyowa_Kirin @orchard_tx #Rare_Diseases
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Gamida Cell Presents Positive Initial Results on Treating Severe Aplastic Anemia (SAA) with Omidubicel - For More Information Visit https://t.co/0l9lwq0ntM
@GamidaCellTx #Severe_Aplastic_Anemia #Rare_Diseases #SAA #Omidubicel #OrphanDrugs #Drug_Research #FDA #Ayrmid
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At SHEIN, we're hoping to build the future of fashion. And our on-demand business model is helping us to reduce waste across our value-chain. Head to the link to learn more about our approach today.
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The EMA Grants Orphan Drug Designation to Neurenati’s NEU-001 for the Treatment of Hirschsprung Disease - For More Information Visit https://t.co/bxTJxk9KwH
@neurenati #Hirschsprung_Disease #Rare_Diseases
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Cabaletta Bio Announces Positive Clinical Data for Rese-Cel Across Multiple Autoimmune Disease Trials at ACR Convergence 2025 - For More Information Visit https://t.co/GFugePGbqV
@CabalettaBio #Rare_Diseases
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GSK’s Antibody-Drug Conjugate GSK’227 Receives EMA Orphan Drug Designation for Pulmonary Neuroendocrine Carcinoma - For More Information Visit https://t.co/qLE4dpoFSq
@GSK #EMA #Rare_Diseases #Lung_Cancer #Pulmonary_Neuroendocrine_Carcinoma #OrphanDrugs #SCLC
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Aviation touches roughly 11% of U.S. GDP in some format, but with today’s air traffic control limitations, it’s also a cap on growth. AI can change that, not by replacing pilots or controllers, but by empowering them with smarter tools that open the skies to many more aircraft.
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Roche Reports Positive Phase III Results for Gazyva/Gazyvaro in Children and Young Adults with Idiopathic Nephrotic Syndrome - For More Information Visit https://t.co/8NYEtQS9GU
@Roche #INS #Rare_Diseases #OrphanDrugs #Idiopathic_Nephrotic_Syndrome #Roche #Drug_Research
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Zenas BioPharma Announces Positive Results from Phase 2 MoonStone Trial of Obexelimab in Relapsing Multiple Sclerosis - For More Information Visit https://t.co/LQUF9mE1Uz
#Zenas_BioPharma #Rare_Diseases #MS #Multiple_Sclerosis #OrphanDrugs #Obexelimab #Drug_research
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BridgeBio Pharma Reports Positive Phase 3 Results for BBP-418 in Limb-Girdle Muscular Dystrophy Type 2I/R9 - For More Information Visit https://t.co/6vTtETCFdM
@BridgeBioPharma #Limb_Girdle #LGMD2I_R9 #Rare_Diseases #Muscular_Dystrophy #OrphanDrugs #Drug_Research
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OncoC4’s Gotistobart Receives Breakthrough Therapy Designation in China for Advanced Squamous NSCLC - For More Information Visit https://t.co/pYVuaS4aZ7
#OncoC4 #NSCLC #Lung_Cancer #Rare_Diseases #OrphanDrugs
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We’re focusing on the “Cactus Creek Book Series” of novels by Janice Cole Hopkins next! Just go to our profile page and feel free to shop from these 5 titles!
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Daewoong’s Bersiporocin Clears Safety Review, Continues Global Phase 2 Trial in Idiopathic Pulmonary Fibrosis (IPF) - For More Information Visit https://t.co/8oiA6c37xJ
#Daewoong #IPF #Rare_Diseases
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FDA Grants Orphan Drug Designation to Zenith Epigenetics’ ZEN-3694 for Rare NUT Carcinoma - For More Information Visit https://t.co/BvpiYFYyRA
#ODD #FDA #NUT_Carcinoma #Cancer #Rare_diseases @zenithepi #OrphanDrugs #Drug_Research #Cancer_research
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FDA Expands Approval of Merck’s WINREVAIR™ for Pulmonary Arterial Hypertension Following Positive Phase 3 ZENITH Trial Results - For More Information Visit https://t.co/ShInuSIb76
@Merck #PAH #Rare_Diseases
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Nipocalimab Shows Significant Reduction in Sjögren’s Disease Activity in Phase 2 DAHLIAS Study Published in The Lancet - For More Information Visit https://t.co/TVJb79r2eO
@JNJInnovMed #Sjögren #Rare_Diseases
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Apotex Receives Health Canada Approval for LOQTORZI®, the First and Only Immunotherapy for Advanced Nasopharyngeal Cancer - For More Information Visit https://t.co/dLAdqXnRzj
#Apotex #Rare_Diseases #Nasopharyngeal_Cancer #OrphanDrugs #Health_Canada
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FDA Grants Breakthrough Therapy Designation to Partner Therapeutics’ Zenocutuzumab-zbco for NRG1+ Advanced Cholangiocarcinoma - For More Information Visit https://t.co/r2kKmISVD0
@PartnerTx #Rare_Diseases #FDA #Cholangiocarcinoma #OrphanDrugs #Drug_Research
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CuraSen Therapeutics Doses First Patient with CuraAX (CST-3056) in Phase 2a Neurogenic Orthostatic Hypotension (nOH) Study - For More Information Visit https://t.co/vhBLjDLQNa
#CuraSen #Rare_Diseases #nOH
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Nuvation Bio Enrolls First Patient in Global Phase 3 Study of Safusidenib for the Treatment of IDH1-Mutant Astrocytoma - For More Information Visit https://t.co/ZNZAr0MnlR
@nuvationbioinc #Brain_Cancer #Rare_Disease #OrphanDrugs #Astrocytoma #Glioma #Drug_Research
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FDA Grants Orphan Drug Designation to Halia Therapeutics’ Ofirnoflast (HT-6184) in the Treatment of Myelodysplastic Syndromes - For More Information Visit https://t.co/KhxeoxHYgM
@Halia_Tx #MDS #Rare_Diseases #FDA #Myelodysplastic_Syndromes #OrphanDrugs #Halia
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Global Study Shows Iza-bren Could Redefine Nasopharyngeal Carcinoma Care with Unprecedented Efficacy - For More Information Visit https://t.co/y8Hc4WjZTR
#Biokin #Nasopharyngeal_Carcinoma #Rare_diseases
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ADARx Pharmaceuticals Begins Phase 3 STOP-HAE Trial and Gains Orphan Drug Status for ADX-324 in Hereditary Angioedema - For More Information Visit https://t.co/oDX2c3vv2e
#Hereditary_Angioedema #ADARx #HAE #Rare_Diseases #OrphanDrugs #Drug_research #Medical_research
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