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62 Newest Publications about the topic hemophilia


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Gene therapies for hemophilia hit the mark in clinical trials

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.


Blood feud erupts over Roche's bispecific antibody for 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


Seroprevalence and clinical characteristics of viral hepatitis in transfusion-dependent thalassemia and hemophilia patients

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 ...


Systemic delivery of factor IX messenger RNA for protein replacement therapy [Medical Sciences]

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 ...


The Utilization of Rehabilitation in Patients with Hemophilia A in Taiwan: A Nationwide Population-Based Study

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 ...


A Cure For Hemophilia: the Promise Becomes a Reality

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


Pharmacokinetics, tissue distribution, excretion, and metabolite profiling of PEGylated rFIX (nonacog beta pegol, N9-GP) in rats

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 ...


Factor VIII associated with lipidic nanoparticles retains efficacy in the presence of anti‐Factor VIII antibodies in Hemophilia A mice

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 ...


[Policy Forum] Paying for future success in gene therapy

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 ...


Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo: delivery vector and immune responses are the key to success

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, ...


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