Last Posted: May 10, 2022
- Haemophilia gene therapy-Update on new country initiatives.
Reiss Ulrike M et al. Haemophilia : the official journal of the World Federation of Hemophilia 2022 28 Suppl 461-67 - Examining patient and professional perspectives in the UK for gene therapy in haemophilia.
Woollacott Ione et al. Haemophilia : the official journal of the World Federation of Hemophilia 2022 - Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A
S Fong et al, Nature Medicine, April 11, 2022 - Changing paradigms of hemophilia care across larger specialized treatment centers in the European region.
Windyga Jerzy et al. Therapeutic advances in hematology 2022 1320406207221088462 - A preliminary application of a haemophilia value framework to emerging therapies in haemophilia.
Skinner Mark W et al. Haemophilia : the official journal of the World Federation of Hemophilia 2022 28 Suppl 29-18 - Is the world ready for gene therapy?
Garrison Louis P et al. Haemophilia : the official journal of the World Federation of Hemophilia 2022 28 Suppl 25-8 - Precision Public Health in Action: Enhancing models to predict risk of adverse treatment outcomes in people with hemophilia
CJ Bean et al, CDC Blog Post, March 29, 2022 - Prepare the Way for Hemophilia A Gene Therapy
CD Thornburg, NEJM, March 17, 2022 - Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A
MC Ozelo et al, NEJM, March 17, 2022 - Managing Severe Hemophilia A in Children: Pharmacotherapeutic Options.
Regling Katherine et al. Pediatric health, medicine and therapeutics 2022 1327-35
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