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Vertex Pharmaceuticals

blood
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory

Zynteglo: Gene Therapy for Beta-thalassemia

September 13, 2022September 13, 2022

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

Trikafta/Kaftrio: World’s First Triple Therapy for Cystic Fibrosis
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory

Trikafta/Kaftrio: World’s First Triple Therapy for Cystic Fibrosis

December 9, 2021September 8, 2022

The combination drug will help almost all cystic fibrosis patients.

blood
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory

Zynteglo: Gene Therapy for Beta-thalassemia

December 4, 2020December 4, 2022

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

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