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62 Newest Publications about the topic hemophiliarss
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07-Feb-2018 | Adrian K Pickar; Charles A Gersbach, Nature Medicine, 2018
Gene therapies for hemophilia hit the mark in clinical trials Gene therapies for hemophilia hit the mark in clinical trials, Published online: 07 February 2018; doi:10.1038/nm.4492 Two recent studies describe clinical successes for single-dose gene therapy in trials for two forms of hemophilia.
11-Sep-2017 | Elie Dolgin, Nature Biotechnology, 2017
Blood feud erupts over Roche's bispecific antibody for hemophilia Nature Biotechnology, Published online: 11 September 2017; doi:10.1038/nbt0917-803
09-Jun-2017 | Tyng-Yuan Jang; Pei-Chin Lin; Ching-I Huang; Yu-Mei Liao; Ming-Lun Yeh; Yu-Sheng Zeng; Po-Cheng Liang; Wan-Yi Hsu; S ..., PLoS ONE, 2017
by Tyng-Yuan Jang, Pei-Chin Lin, Ching-I Huang, Yu-Mei Liao, Ming-Lun Yeh, Yu-Sheng Zeng, Po-Cheng Liang, Wan-Yi Hsu, Shih-Pien Tsai, Zu-Yau Lin, Shinn-Cherng Chen, Jee-Fu Huang, Chia-Yen Dai, Chung-Feng Huang, Shyh-Shin Chiou, Wan-Long Chuang, Ming-Lung Yu Background/Aims Transfusion dependent ...
07-Mar-2017 | Suvasini Ramaswamy; Nina Tonnu; Kiyoshi Tachikawa; Pattraranee Limphong; Jerel B. Vega; Priya P. Karmali; Pad Chivuk ..., Proceedings of the National Academy of Sciences current issue, 2017
Safe and efficient delivery of messenger RNAs for protein replacement therapies offers great promise but remains challenging. In this report, we demonstrate systemic, in vivo, nonviral mRNA delivery through lipid nanoparticles (LNPs) to treat a Factor IX (FIX)-deficient mouse model of hemophilia ...
30-Sep-2016 | Chien-Min Chen; Yao-Hsu Yang; Chia-Hao Chang; Chih-Cheng Chen; Pau-Chung Chen, PLoS ONE, 2016
by Chien-Min Chen, Yao-Hsu Yang, Chia-Hao Chang, Chih-Cheng Chen, Pau-Chung Chen Introduction Rehabilitation plays an important role in the physical health of patients with hemophilia. However, comprehensive information regarding the utilization of rehabilitation for such patients remains ...
01-Sep-2016 | Roland W Herzog, Molecular Therapy, 2016
A Cure For Hemophilia: the Promise Becomes a Reality Molecular Therapy 24, 1503 (september 2016). doi:10.1038/mt.2016.169 Author: Roland W Herzog
14-Aug-2016 | Author(s): Ola Sternebring, Jesper Kammersgaard Christensen, Inga Bjørnsdottir, European Journal of Pharmaceutical Science, 2016
Publication date: 20 September 2016 Source:European Journal of Pharmaceutical Sciences, Volume 92 Author(s): Ola Sternebring, Jesper Kammersgaard Christensen, Inga Bjørnsdottir Nonacog beta pegol (N9-GP) is a novel recombinant factor IX conjugated with a 40-kDa branched polyethylene ...
15-Jul-2016 | Krithika A. Shetty, Matthew P. Kosloski, Donald E. Mager, Sathy V. Balu‐Iyer, Biopharmaceutics & Drug Disposition, 2016
Abstract Development of inhibitory antibodies against Factor VIII (FVIII) is a major challenge in Hemophilia A (HA) therapy. Such antibodies develop in nearly 30% of the patients receiving replacement FVIII, abrogating therapeutic efficacy. In this work, we evaluated whether B‐domain deleted ...
27-May-2016 | Stuart H. Orkin; Philip Reilly, Science , 2016
Imagine a young man with hemophilia A who no longer has to self-administer factor VIII replacement; an individual with sickle cell disease who is free of chronic pain and intermittent crises; a girl functionally blind since the age of 5 who can now see; or a baby rescued from a fatal, inherited ...
04-Apr-2016 | Tuan Huy Nguyen, Ignacio Anegon, EMBO Molecular Medicine, 2016
Hemophilia B is a serious hemostasis disorder due to mutations of the factor IX gene in the X chromosome. Gene therapy has gained momentum in recent years as a therapeutic option for hemophilia B. In hemophilia, reconstitution with a mere 1–2% of the clotting factor improves the quality of life, ...