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If approved, Pfizer's hemophilia A gene therapy will compete with BioMarin Pharmaceutical's one-time treatment Roctavian, which was approved in the U.S. last year and is priced at $2.9 million.
U.S. officials on Thursday approved drugmaker BioMarin's gene therapy for the most common form of hemophilia, a $2.9 million infused treatment that can significantly reduce dangerous bleeding ...
Roctavian is the first gene replacement therapy for the most common form of hemophilia, enabling patients a way to forego or reduce the need for lifetime treatment with factor proteins needed to ...
Valoctocogene roxaparvovec, sold under the brand name Roctavian, is a gene therapy used for the treatment of hemophilia A. [ 1 ] [ 3 ] It was developed by BioMarin Pharmaceutical . [ 4 ] [ 5 ] [ 6 ] Valoctocogene roxaparvovec is made of a virus (AAV5) that has been modified to contain the gene for factor VIII, which is lacking in people with ...
Etranacogene dezaparvovec, sold under the brand name Hemgenix is a gene therapy used for the treatment of hemophilia B. [5] [6] [7] Etranacogene dezaparvovec is an adeno-associated virus vector-based gene therapy which consists of a viral vector carrying a gene for clotting Factor IX. [7]
The best results have been found in haemophilia B. [88] In 2016 early stage human research was ongoing with a few sites recruiting participants. [88] In 2017 a gene therapy trial on nine people with haemophilia A reported that high doses did better than low doses. [89] [90] It is not currently an accepted treatment for haemophilia. [16]
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