Results 81 to 90 of about 13,000,271 (286)

Patient and parent preferences for characteristics of prophylactic treatment in hemophilia

Patient Preference and Adherence, 2015
Roberto Furlan,1 Sangeeta Krishnan,2 Jeffrey Vietri3 1Advanced Methods, Kantar Health, Epsom, Surrey, UK; 2Global Health Economics and Outcomes Research, Biogen, MA, USA; 3Health Outcomes, Kantar Health, Milan, Italy Introduction: New longer-acting ...
Furlan R, Krishnan S, Vietri J
doaj  

Real-World Analysis of Dispensed IUs of Coagulation Factor IX and Resultant Expenditures in Hemophilia B Patients Receiving Standard Half-Life Versus Extended Half-Life Products and Those Switching from Standard Half-Life to Extended Half-Life Products

Journal of Managed Care & Specialty Pharmacy, 2018
BACKGROUND: Hemophilia B requires replacement therapy with factor IX (FIX) coagulation products to treat and prevent bleeding episodes. A recently introduced extended half-life (EHL) recombinant FIX replacement product provided the opportunity to compare
B. Tortella, J. Alvir, Margaret V. McDonald, D. Spurden, P. Fogarty, A. Chhabra, A. Pleil   +6 more
semanticscholar   +1 more source

Very low dislocation rate and good clinical outcome after Bereiter trochleoplasty and additional procedures following the Copenhagen patella–femoral instability algorithm: One‐ and two‐years outcomes from a consecutive cohort of 368 cases

Knee Surgery, Sports Traumatology, Arthroscopy, EarlyView.
Abstract Purpose Patello–femoral instability (PFI) is often caused by predisposing factors, with trochlea dysplasia (TD) as the most prominent. Untreated patellar instability leads to impaired function and an increased risk of patellofemoral osteoarthritis.
Christian Dippmann, Volkert Siersma, Simone Rechter, Kristoffer W. Barfod, Peter Lavard   +4 more
wiley   +1 more source

Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models

Journal of Translational Medicine, 2017
Background Adeno-associated virus (AAV) gene therapy vectors have shown the best outcomes in human clinical studies for the treatment of genetic diseases such as hemophilia.
David M. Markusic, Timothy C. Nichols, Elizabeth P. Merricks, Brett Palaschak, Irene Zolotukhin, Damien Marsic, Sergei Zolotukhin, Arun Srivastava, Roland W. Herzog   +8 more
doaj   +1 more source

Optimising outcomes in lateral unicompartmental knee arthroplasty: Analysing 25 years of registry data

Knee Surgery, Sports Traumatology, Arthroscopy, EarlyView.
Abstract Purpose Limited data and experience surround lateral unicompartmental knee arthroplasty (UKA), contributing to uncertainty regarding its broader application in clinical practice. To understand how to optimise lateral UKA outcomes, this study aimed to evaluate the implant survival of lateral UKA and compare it to total knee arthroplasty (TKA ...
Kristine Ifigenia Bunyoz, Martin Lindberg‐Larsen, Kirill Gromov, Anders Troelsen   +3 more
wiley   +1 more source

Targeted genome engineering in human induced pluripotent stem cells from patients with hemophilia B using the CRISPR-Cas9 system

Stem cell research & therapeutics, 2018
BackgroundReplacement therapy for hemophilia remains a lifelong treatment. Only gene therapy can cure hemophilia at a fundamental level. The clustered regularly interspaced short palindromic repeats–CRISPR associated nuclease 9 (CRISPR-Cas9) system is a ...
C. Lyu, Jun Shen, Rui Wang, Haihui Gu, Jian-Ping Zhang, F. Xue, Xiaofan Liu, Wei Liu, R. Fu, Liyan Zhang, Huiyuan Li, Xiaobing Zhang, Tao Cheng, Renchi Yang, Lei Zhang   +14 more
semanticscholar   +1 more source

Antithrombin: Deficiency, Diversity, and the Future of Diagnostics

Mass Spectrometry Reviews, EarlyView.
ABSTRACT Our healthcare system provides reactive sick‐care, treating patients after symptoms have appeared by prescription of generic and often suboptimal therapy. This strategy brings along high costs and high pressure which is not sustainable.
Mirjam Kruijt, Christa M. Cobbaert, L. Renee Ruhaak   +2 more
wiley   +1 more source

Mild-severe hemophilia B impacts relationships of US adults and children with hemophilia B and their families: results from the B-HERO-S study

Patient Related Outcome Measures, 2019
Susan Cutter,1 Christine Guelcher,2 Susan Hunter,3 Dawn Rotellini,4 Spencer Dunn,5 David L Cooper61Penn Comprehensive Hemophilia and Thrombosis Program, Hospital of the University of Pennsylvania, Philadelphia, PA, USA; 2Comprehensive Hemostasis and ...
Cutter S, Guelcher C, Hunter S, Rotellini D, Dunn S, Cooper DL   +5 more
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Reducing Surgery for Pediatric Posttonsillectomy Hemorrhage Using Tranexamic Acid: A Quality Improvement Initiative

Otolaryngology–Head and Neck Surgery, EarlyView.
Abstract Objective Evaluate the use of tranexamic acid (TXA) and observation as a management option for pediatric patients presenting with posttonsillectomy hemorrhage (PTH). Study Design Retrospective analysis of a prospectively implemented quality improvement initiative with a historical control comparison group. Setting Tertiary children's hospital.
Laura A. Petrauskas, Janavi Sethurathnam, Ansley J. Kunnath, Rahul K. Sharma, John Ceremsak, Ryan H. Belcher, James D. Phillips, Jay A. Werkhaven, Amy S. Whigham, Lyndy J. Wilcox, Christopher T. Wootten, Frank W. Virgin, Jason S. Park   +12 more
wiley   +1 more source

Autologous and Heterologous Cell Therapy for Hemophilia B toward Functional Restoration of Factor IX

Cell Reports, 2018
Summary: Hemophilia B is an ideal target for gene- and cell-based therapies because of its monogenic nature and broad therapeutic index. Here, we demonstrate the use of cell therapy as a potential long-term cure for hemophilia B in our FIX-deficient ...
Suvasini Ramaswamy, Nina Tonnu, Tushar Menon, Benjamin M. Lewis, Kevin T. Green, Derek Wampler, Paul E. Monahan, Inder M. Verma   +7 more
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