Tebentafusp is a pharmacological breakthrough in the fight against a deadly disease.
Rare/Orphan Diseases
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 inMetabolism, 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.
Posted inAutoimmune Diseases, New Drugs, Rare/Orphan Diseases, Regulatory
Vyvgart: New Drug to Treat Myasthenia Gravis
Efgartigimod developed by Argenx is a strong competitor to Soliris.
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory
Trikafta/Kaftrio: World’s First Triple Therapy for Cystic Fibrosis
The combination drug will help almost all cystic fibrosis patients.
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory
Voxzogo: First Drug to Treat Dwarfism
Vosoritide by BioMarin Pharmaceutical may cure achondroplasia.
