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[@CheckOrphan](/creator/twitter/CheckOrphan)
"Akeso Announces First Patient Dose in Global Registrational Trial of Cadonilimab (PD-1/CTLA-4) for PD-1 Treatment-Resistant Hepatocellular Carcinoma - For More Information Visit @AkesoInc #HCC #Rare_Diseases #OrphanDrugs #Hepatocellular_Carcinoma"  
[X Link](https://x.com/CheckOrphan/status/1967594751198462334) [@CheckOrphan](/creator/x/CheckOrphan) 2025-09-15T14:21Z 18.1K followers, XXX engagements


"Ono Pharmaceutical Reports Positive Interim Phase X Results for ONO-2808 in Multiple System Atrophy (MSA) - For More Information Visit @Ono_pharma_PR #Multiple_System_Atrophy #MSA #Rare_Diseases #OrphanDrugs #Drug_Research #Ono_pharma"  
[X Link](https://x.com/CheckOrphan/status/1976653704112771086) [@CheckOrphan](/creator/x/CheckOrphan) 2025-10-10T14:19Z 18.1K followers, XXX engagements


"Genmab Reports Updated Phase X Results Supporting Outpatient Use of Epcoritamab in R/R Diffuse Large B-Cell Lymphoma - For More Information Visit #Diffuse_Large_B_Cell_Lymphoma #DLBCL #Rare_Diseases #OrphanDrugs #Drug_Research @Genmab"  
[X Link](https://x.com/CheckOrphan/status/1963233516717502590) [@CheckOrphan](/creator/x/CheckOrphan) 2025-09-03T13:32Z 18.1K followers, XXX engagements


"Dyne Therapeutics Receives Orphan Drug Designation in Japan for DYNE-251 in Duchenne Muscular Dystrophy - For More Information Visit @Dyne_tx #Duchenne #Muscular_Dystrophy #Rare_Diseases"  
[X Link](https://x.com/CheckOrphan/status/1972700098527117316) [@CheckOrphan](/creator/x/CheckOrphan) 2025-09-29T16:28Z 18.1K followers, XXX engagements


"AbbVie Submits Biologics License Application to FDA for Pivekimab Sunirine (PVEK) in Rare Blood Cancer BPDCN - For More Information Visit @abbvie #Rare_diseases #Blood_Cancer #BPDCN"  
[X Link](https://x.com/CheckOrphan/status/1973392385963876394) [@CheckOrphan](/creator/x/CheckOrphan) 2025-10-01T14:19Z 18.1K followers, XXX engagements


"Taysha Gene Therapies Announces FDA Breakthrough Therapy Designation and Provides Positive Regulatory Update on TSHA-102 in Rett Syndrome - For More Information Visit @TayshaGTx #Rare_Diseases #OrphanDrugs #FDA #Rett_Syndrome #Drug_research"  
[X Link](https://x.com/CheckOrphan/status/1973755779154067471) [@CheckOrphan](/creator/x/CheckOrphan) 2025-10-02T14:23Z 18.1K followers, XXX engagements


"Dyne Therapeutics Reports One-Year Data Showing Meaningful Functional Gains with Z-Basivarsen in Myotonic Dystrophy Type X (DM1) - For More Information Visit @Dyne_tx #DM1 #Rare_Diseases #Myotonic_Dystrophy #OrphanDrugs #Drug_research"  
[X Link](https://x.com/CheckOrphan/status/1975579659309121583) [@CheckOrphan](/creator/x/CheckOrphan) 2025-10-07T15:11Z 18.1K followers, XXX engagements


"NEJM Publishes Study Showing How Regenerons DB-OTO Restored Hearing in XX of XX Children with Profound Genetic Hearing Loss - For More Information Visit @Regeneron #Hearing_Loss #FDA #NEJM #Rare_Diseases #OrphanDrugs #Pediatrics"  
[X Link](https://x.com/CheckOrphan/status/1977716751488929953) [@CheckOrphan](/creator/x/CheckOrphan) 2025-10-13T12:43Z 18.1K followers, XXX engagements


"argenx Announces Positive Topline Results from ADAPT SERON Study of VYVGART in Patients with AChR-Ab Seronegative gMG - For More Information Visit @argenxglobal #MyastheniaGravis #FDA #Rare_Diseases #OrphanDrugs #Drug_Discovery #VYVGART"  
[X Link](https://x.com/CheckOrphan/status/1959984592934326657) [@CheckOrphan](/creator/x/CheckOrphan) 2025-08-25T14:21Z 18.1K followers, XXX engagements


"Ipsen Wins Japanese Approval for Bylvay to Treat Pruritus in Children with Rare Liver Disorder Progressive Familial Intrahepatic Cholestasis (PFIC) - For More Information Visit #Rare_diseases #PFIC #progressive_Familial_Intrahepatic_Cholestasis"  
[X Link](https://x.com/CheckOrphan/status/1969035875955699925) [@CheckOrphan](/creator/x/CheckOrphan) 2025-09-19T13:48Z 18.1K followers, XXX engagements


"Neurocrine Biosciences: INGREZZA Achieves Robust Symptomatic Remission in Tardive Dyskinesia Patients in KINECT-PRO Phase X Study - For More Information Visit @neurocrine #INGREZZA #Rare_Diseases #OrphanDrugs #Tardive_Dyskinesia #Drug_Research"  
[X Link](https://x.com/CheckOrphan/status/1970473915983225323) [@CheckOrphan](/creator/x/CheckOrphan) 2025-09-23T13:02Z 18.1K followers, XXX engagements


"CSL Vifor and Travere Therapeutics Highlight Inclusion of FILSPARI in Updated 2025 KDIGO Guidelines for IgA Nephropathy Treatment - For More Information Visit @CSL @TravereRare #Rare_Diseases #OrphanDrugs #IgANephropathy #IgAN #Drug_research"  
[X Link](https://x.com/CheckOrphan/status/1971555201527287894) [@CheckOrphan](/creator/x/CheckOrphan) 2025-09-26T12:39Z 18.1K followers, XXX engagements


"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 @novonordisk #Hemophilia #Rare_diseases"  
[X Link](https://x.com/CheckOrphan/status/1972668872164606161) [@CheckOrphan](/creator/x/CheckOrphan) 2025-09-29T14:24Z 18.1K followers, XXX engagements


"Neurocrine Biosciences Reports Three-Year Data Showing Sustained Chorea Improvement and Long-Term Safety of INGREZZA in Huntingtons Disease - For More Information Visit @neurocrine #HuntingtonDisease #Rare_Diseases #OrphanDrugs"  
[X Link](https://x.com/CheckOrphan/status/1975195704688787610) [@CheckOrphan](/creator/x/CheckOrphan) 2025-10-06T13:45Z 18.1K followers, XXX engagements


"FDA Approves Regenerons Libtayo as First Adjuvant Immunotherapy for High-Risk Cutaneous Squamous Cell Carcinoma (CSCC) - For More Information Visit @Regeneron #CSCC #Rare_Diseases #FDA #Squamous_Cell_Carcinoma #OrphanDrugs #Libtayo"  
[X Link](https://x.com/CheckOrphan/status/1976257861287043215) [@CheckOrphan](/creator/x/CheckOrphan) 2025-10-09T12:06Z 18.1K followers, XXX engagements