Valoctocogene roxaparvovec: first approval
HA Blair - Drugs, 2022 - Springer
Valoctocogene roxaparvovec (ROCTAVIAN™) is a gene therapy being developed by
BioMarin Pharmaceutical Inc. for the treatment of haemophilia A. In August 2022 …
BioMarin Pharmaceutical Inc. for the treatment of haemophilia A. In August 2022 …
[HTML][HTML] The ice age-a review on formulation of adeno-associated virus therapeutics
P Grossen, IS Koukelli, J van Haasteren… - European Journal of …, 2023 - Elsevier
Gene therapies offer promising therapeutic alternatives for many disorders that currently lack
efficient treatment options. Due to their chemical nature and physico-chemical properties …
efficient treatment options. Due to their chemical nature and physico-chemical properties …
Blood biodistribution and vector shedding of valoctocogene roxaparvovec in people with severe hemophilia A
S Agarwal, K Sandza, K Obrochta Moss… - Blood …, 2024 - ashpublications.org
Following systemically administered adeno-associated virus gene therapy, vector particles
are widely distributed, raising concerns about horizontal or germline vector transmission …
are widely distributed, raising concerns about horizontal or germline vector transmission …
Gene therapy in hemophilia: the dawn of a new era
Hemophilia A and hemophilia B are hereditary bleeding disorders associated with the X
chromosome, stemming from genetic defects in the coding of coagulation Factor VIII (FVIII) …
chromosome, stemming from genetic defects in the coding of coagulation Factor VIII (FVIII) …
[HTML][HTML] Therapeutic Application and Structural Features of Adeno-Associated Virus Vector
Y Matsuzaka, R Yashiro - Current Issues in Molecular Biology, 2024 - mdpi.com
Adeno-associated virus (AAV) is characterized by non-pathogenicity, long-term infection,
and broad tropism and is actively developed as a vector virus for gene therapy products …
and broad tropism and is actively developed as a vector virus for gene therapy products …
Valoctocogene Roxaparvovec
TL Levien, DE Baker - Hospital Pharmacy, 2024 - journals.sagepub.com
Each month, subscribers to The Formulary Monograph Service receive 5 to 6 well-
documented monographs on drugs that are newly released or are in late phase 3 trials. The …
documented monographs on drugs that are newly released or are in late phase 3 trials. The …