One dose of betibeglogene autotemcel will permanently eliminate the dependence on regular blood transfusions.
CRISPR Therapeutics
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 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.