Results 61 to 70 of about 50,045 (313)

Cost‐effectiveness of fenfluramine as add‐on treatment in the management of Dravet Syndrome: A real‐world multicenter study

Epilepsia Open, EarlyView.
Abstract Objective Dravet syndrome (DS) is a rare disease with a high clinical and socioeconomic impact on patients, society, and the healthcare system. The recent approval of therapies such as fenfluramine (FFA) has transformed the treatment landscape; however, data on their cost‐effectiveness are still scarce. This study evaluates the real‐world cost‐
Paolo A. Cortesi, Carla Fornari, Alessandra Boncristiano, Rita Facchetti, Simona Balestrini, Viola Doccini, Nicola Specchio, Nicola Pietrafusa, Marina Trivisano, Francesca Darra, Alberto Cossu, Domenica I. Battaglia, Michela Quintiliani, Maria L. Gambardella, Lorenzo G. Mantovani, Renzo Guerrini   +15 more
wiley   +1 more source

POLYMORPHISM OF THROMBOPHILIA GENES AND THEIR ROLE IN DEVELOPMENT OF DIFFERENT DISEASE PHENOTYPES AND THROMBOTIC COMPLICATIONS IN HEMOPHILIA PATIENTS

Сибирский научный медицинский журнал, 2019
Objective was to study genetic markers of thrombophilia in patients with hemophilia, which can affect the course of the disease and contribute to thrombotic complications. Material and methods.
T. Yu. Polyanskaya, I. S. Fevraleva, E. E. Karpov, N. V. Sadykova, M. S. Sampiev, A. V. Golobokov, G. V. Mishin, D. Yu. Petrovskiy, A. A. Koroleva, G. M. Galstyan, A. B. Sudarikov, V. Yu. Zorenko   +11 more
doaj   +1 more source

Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques [PDF]

, 2002
The feasibility, safety, and efficacy of liver-directed gene transfer was evaluated in 5 male macaques (aged 2.5 to 6.5 years) by using a recombinant adeno-associated viral (rAAV) vector (rAAV-2 CAGG-hFIX) that had previously mediated persistent ...
Davidoff, AM, Hanawa, H, Hoffer, FA, Hu, Y, Lozier, JN, Mandrell, TD, Nathwani, AC, Ng, CYC, Nienhuis, AW, Nikanorov, A, Slaughter, C, Vanin, EF, Zhou, J   +12 more
core   +1 more source

Christmas Disease (Hemophilia –B) – A Case Report

Bangabandhu Sheikh Mujib Medical University Journal, 2010
We report a 25 years old man developed Haemarthrosis of left hip joint with a history of recurrent swelling and pain in multiple joints and prolonged bleeding following minor trauma since childhood.
Md Rafiqul Alam, Mohammad Saiful Habib, Mohammad Arifur Rahman, Md Mizanur Rahman Khan, MA Jalil Chowdhury, Taimur AK Mahmud   +5 more
doaj   +1 more source

A novel knee implant for total knee arthroplasty meets expectations at 10 years. First long‐term follow‐up report of clinical outcomes and survivorship

Knee Surgery, Sports Traumatology, Arthroscopy, EarlyView.
Abstract Purpose Achieving a “forgotten knee” after total knee arthroplasty (TKA) remains a primary goal in modern knee replacement surgery. Anatomic implant designs aim to replicate native knee anatomy and kinematics, potentially improving patient satisfaction and functional outcomes.
Alice Montagna, Marina Marescalchi, Virginia Cinelli, Rudy Sangaletti, Luca Andriollo, Francesco Benazzo, Stefano Marco Paolo Rossi   +6 more
wiley   +1 more source

Cyclosporine treatment of acquired hemophilia due to factor VIII antibodies [PDF]

, 2000
Acquired hemophilia, caused by autoantibodies against coagulation factor VIII, is usually treated with steroids, cyclophosphamide, intravenous gammaglobulins and sporadically other drugs.
Baele, Gaston, Derom, Eric, Petrovic, Mirko   +2 more
core  

Chemical Chaperones Improve Protein Secretion and Rescue Mutant Factor VIII in Mice with Hemophilia A. [PDF]

, 2012
nefficient intracellular protein trafficking is a critical issue in the pathogenesis of a variety of diseases and in recombinant protein production.
Abriss, Daniela, Milanov, Peter, Pepperkok, Rainer, Quade-Lyssy, Patricia, Roth, Stefanie D., Schüttrumpf, Jörg, Seifried, Erhard, Simpson, Jeremy C., Tonn, Torsten, Ungerer, Christopher   +9 more
core   +2 more sources

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

Could PTH/Ca Ratio Serve as a New Marker for Evaluating Bone Metabolism in Hemophilia Patients?

Diagnostics
Background/Objectives: Low bone mineral density (BMD) is common in hemophilia patients. Identifying high-risk patients for low BMD early is essential to prevent complications and reduce morbidity.
Tuba Ersal, Fazıl Çağrı Hunutlu, Vildan Gürsoy, Ezel Elgün, Şeyma Yavuz, İpek Dal Akkuş, İlayda Baş, Vildan Özkocaman, Fahir Özkalemkaş   +8 more
doaj   +1 more source

New Technologies Empower Hemophilic Arthropathy Treatment: Focusing on Regenerative Medicine, Molecular Targeting, and Gene Therapy

Medicine Bulletin, EarlyView.
ABSTRACT Hemophilic arthropathy (HA), a major complication of hemophilia, is a chronic osteoarthropathy driven by repeated joint bleeding. Although traditional therapies such as coagulation factor replacement, physical rehabilitation, and surgery can manage symptoms, they cannot fundamentally repair established joint damage or break the pathological ...
Lin Sun, Yingnan Wang, Leyuan Zhao, Liqiang Lu, Fushan Hou, Chuan Yin, Kun Zhang, Yongqiang Hao, Jingke Fu   +8 more
wiley   +1 more source