Redasemtide as a lifesaver for the most devastating diseases, including stroke, osteoarthritis, cardiomyopathy, and bullous epidermolysis.
Rare/Orphan Diseases
Posted inAutoimmune Diseases, New Drugs, Rare/Orphan Diseases, Regulatory
Enjaymo: First Drug to Treat Cold Agglutinin Disease
Sutimlimab will reduce the need for red blood cell transfusion due to hemolysis.
Posted inAutoimmune Diseases, New Drugs, Rare/Orphan Diseases, Regulatory
Tavneos: New Drug for ANCA-Associated Vasculitis
Avacopan is better than prednisone in the treatment of granulomatosis with polyangiitis and microscopic polyangiitis.
Posted inNew Drugs, Oncology, Rare/Orphan Diseases, Regulatory
Kimmtrak: Groundbreaking Immuno-Oncological Treatment for Metastatic Uveal Melanoma
Tebentafusp is a pharmacological breakthrough in the fight against a deadly disease.
Posted inNew Drugs, Oncology, Rare/Orphan Diseases, Regulatory
Welireg: New Treatment for Tumors in Von Hippel–Lindau Disease
Belzutifan by Merck & Co. is for the treatment of renal cell carcinoma, central nervous system hemangioblastomas, and pancreatic neuroendocrine tumors.
Posted inNeuroscience, Pharma & Biotech, Rare/Orphan Diseases
Biogen to Develop Updated Version of Spinraza
Nusinersen for the treatment of spinal muscular atrophy will get a follow-up.
Posted inCardiology, Metabolism, New Drugs, Rare/Orphan Diseases, Regulatory
Leqvio: Newest Drug Against ‘Very Bad’ Cholesterol
Two injections of inclisiran a year and you can forget about high cholesterol. Novartis doesn’t lie.