One dose of betibeglogene autotemcel will permanently eliminate the dependence on regular blood transfusions.
Rare/Orphan Diseases
Posted inNew Drugs, Oncology, Rare/Orphan Diseases, Regulatory
Tecvayli: New Drug for Multiple Myeloma
Teclistamab by Johnson & Johnson is a new hope for refractory or relapsing multiple myeloma.
Posted inClinical Trials, Neuroscience, Rare/Orphan Diseases
Qalsody: New Drug for Amyotrophic Lateral Sclerosis with SOD1 Mutation
Tofersen and the mixed results of treatment for the deadly neurodegenerative disease.
Posted inClinical Trials, Neuroscience, Rare/Orphan Diseases
Alsitek: New Drug for ALS
Oral masitinib doubles the life in patients with amyotrophic lateral sclerosis.
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.