Oral masitinib doubles the life in patients with amyotrophic lateral sclerosis.
Rare/Orphan Diseases
Posted inClinical Trials, Infections, Rare/Orphan Diseases
Olorofim: New Antifungal Drug
Olorophim by F2G and Shionogi & Co. is focused on invasive aspergillosis treatment.
Posted inAutoimmune Diseases, New Drugs, Rare/Orphan Diseases, Regulatory
Tepezza: First Drug for Thyroid Eye Disease
Teprotumumab by Horizon Therapeutics can really help with proptosis and diplopia in Graves’ disease and beyond.
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory
Vijoice: First Targeted Treatment for PIK3CA-Related Overgrowth Spectrum (PROS)
Novartis and its alpelisib will help with very rare diseases with malformations and excessive tissue growth.
Posted inClinical Trials, Metabolism, Neuroscience, Rare/Orphan Diseases
Redasemtide: Universal Medicine for Regeneration of Any Tissue
Redasemtide as a lifesaver for the most devastating diseases, including stroke, osteoarthritis, cardiomyopathy, and bullous epidermolysis.
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.
