One dose of betibeglogene autotemcel will permanently eliminate the dependence on regular blood transfusions.
Vertex Pharmaceuticals
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
Zynteglo: Gene Therapy for Beta-thalassemia
One dose of betibeglogene autotemcel will permanently eliminate the dependence on regular blood transfusions.
