Once-Weekly TransCon® CNP (Navepegritide) Receives Positive CHMP Opinion for the Treatment of Achondroplasia in Children - For More Information Visit tinyurl.com/4n6sa75f @AscendisPharma #CHMP #EMA #Rare_Diseases #Achondroplasia #Orphan_Drugs

Propanc Biopharma Reports Promising Preclinical Findings for PRP in Overcoming Chemotherapy Resistance in Pancreatic Cancer - For More Information Visit tinyurl.com/3j23ap7u @PropancBio #Rare_Diseases #Orphan_Drugs #Pancreatic_Cancer #Propanc #Cancer_Research

Sobi Receives Positive CHMP Opinion for TRYNGOLZA® (olezarsen) in the Treatment of Severe Hypertriglyceridemia (sHTG) - For More Information Visit tinyurl.com/28ra7zpm #Sobi #sHTG #Rare_Diseases #CHMP #TRYNGOLZA #Severe_Hypertriglyceridemia #Orphan_Drugs

FDA Approves Roche’s Enspryng, a First-in-Class IL-6R Inhibitor, as the First and Only at-home Subcutaneous Treatment Option for Thyroid Eye Disease - For More Information Visit tinyurl.com/34ms3pbj @Roche #Thyroid_Eye_Disease #Eye_Diseases #Rare_Diseases #Orphan_Drugs

Egetis Therapeutics Announces Publication of Largest Retrospective Study to Date on Tiratricol Treatment in Resistance to Thyroid Hormone Beta - For More Information Visit tinyurl.com/2tr79ez9 @Egetis_EGTX #RTH_beta #Resistance_to_Thyroid_Hormone_Beta

Opus Genetics Presents Positive Clinical Data from OPGx-BEST1 and OPGx-LCA5 Gene Therapy Programs at Retina in Rio 2026 - For More Information Visit tinyurl.com/mr4y5x8u #Opus_Genetics #Rare_Diseases #LCA5 #Orphan_Drugs #Leber_Congenital_Amaurosis #Eye_Disorders

Palvella Therapeutics to Present New Pediatric Analyses of QTORIN™ Rapamycin for Microcystic Lymphatic Malformations and cVMs at PeDRA Annual Conference - For More Information Visit tinyurl.com/t9c3adfv @PalvellaTX #Microcystic_Lymphatic_Malformations #cVMs

Alyftrek May Reverse Irreversible Pancreatic Damage in Young Children With Cystic Fibrosis, Reducing Need for Enzyme Replacement Therapy - For More Information Visit tinyurl.com/2shcs6bc @VertexPharma #CF #Cystic_Fibrosis #Alyftrek #Rare_Diseases #Orphan_Drugs

Krystal Biotech Announces Positive Clinical Data for KB113, Demonstrating Basal Cell Carcinoma Regression in Gorlin Syndrome Patients - For More Information Visit tinyurl.com/2cua2aam @KrystalBiotech #Rare_diseases #Gorlin_Syndrome #Orphan_Drugs #Carcinoma

Spinal Muscular Atrophy: New Treatment Options Promote Greater Independence and Quality of Life - For More Information Visit tinyurl.com/2zfc4rr9 #SMA #Spinal_Muscular_Atrophy #Rare_Diseases

Sarepta Reports Preclinical Data Showing SRP-1003 Achieves Up to 80% Nuclear and Cytoplasmic DMPK Knockdown in Myotonic Dystrophy Type 1 - For More Information Visit tinyurl.com/yds3my9a @Sarepta #DM1 #Myotonic_Dystrophy #Rare_Diseases #Orphan_Drugs

Propanc Biopharma Announces Promising Pilot Study Results Demonstrating Potent Anti-Tumor Efficacy of PRP in Pancreatic and Ovarian Cancer - For More Information Visit tinyurl.com/4vm2nrc6 #Propanc #Ovarian_Cancer #Pancreatic_Cancer #Rare_Diseases #Orphan_Drugs

Quoin Pharmaceuticals Announces European Medicines Agency Grants Orphan Drug Designation for QRX003 in the Treatment of Peeling Skin Syndrome - For More Information Visit tinyurl.com/2wz68k2a @QuoinPharma #Peeling_Skin_Syndrome #Rare_Diseases #Orphan_Drugs

Pharming Presents Positive Phase II Topline Data for Leniolisib in Genetic PIDs with Immune Dysregulation at ESID 2026 - For More Information Visit tinyurl.com/4p72ufsx @PharmingGroupNV #Rare_Diseases #PIDs #Orphan_Drugs #Primary_Immunodeficiencies #Drug_research

Trikafta Improves Work Productivity and Daily Activities in People With Cystic Fibrosis, Canadian Study Finds - For More Information Visit tinyurl.com/ycxb2ks3 #Cystic_Fibrosis #Rare_Diseases #Orphan_Drugs #CF

First Cohort Under Amended Protocol Succesfully Completes Safety Evaluation In HOVON 172 MF Clinical Study Of Tasquinimod In Myelofibrosis - For More Information Visit tinyurl.com/5atca9n6 #Active_Biotech #Myelofibrosis #Rare_Diseases #Orphan_Drugs #Tasquinimod

aTyr Pharma Announces Publication Demonstrating Anti-Fibrotic Effects of ATYR0101 in the Journal of Biological Chemistry - For More Information Visit tinyurl.com/39643yj9 @aTyrNews #Fibrotic_Diseases #Fibrosis #IPF #Peyronie_Disease #Rare_Diseases #Dupuytren

Regeneron’s Pasatru Shows Sustained Efficacy in Reducing New Bone Growth and Disease Flare-Ups in Adults With FOP at 84 Weeks - For More Information Visit tinyurl.com/3xhpj8v4 @Regeneron #Rare_Diseases #FOP #Fibrodysplasia_Ossificans_Progressiva #Orphan_Drugs

Anixa Biosciences' Ovarian Cancer CAR-T Therapy Inventor to Deliver Keynote at OC Midwest Focus Conference - For More Information Visit tinyurl.com/y39bzfpc @AnixaBio #Ovarian_Cancer @MayoClinic

Mirum Pharmaceuticals Announces Primary Endpoint Met in Phase 3 EXPAND Study of LIVMARLI® (maralixibat) in Ultra-Rare Cholestatic Liver Diseases - For More Information Visit tinyurl.com/y7zpwcwz @mirumpharma #Cholestatic_Liver_Diseases #Rare_Diseases #Orphan_Drugs