Results 61 to 70 of about 69,645 (303)

Molecular Study of Occult Hepatitis C Infection among Iranian Hemophilia Patients Treated with Direct-acting Antiviral Agents

Journal of Medical Microbiology and Infectious Diseases, 2020
Introduction: A new pathological form of HCV named as occult HCV infection (OCI) has been recently characterized by the presence of HCV RNA in liver biopsy and/or peripheral blood mononuclear cell specimens (PBMCs) and the absence of detectable ...
Elnaz Agi, Saber Asghari, Ali Namvar, Niloofar Khairkhah, Niloofar Naderi, Ali Anvar, Alireza Azizi Saraji, Azam Bolhassani   +7 more
doaj  

An international registry of patients with plasminogen deficiency (HISTORY)

Haematologica, 2020
Plasminogen deficiency is an ultra-rare multisystem disorder characterized by the development of fibrin-rich pseudomembranes on mucous membranes. Ligneous conjunctivitis, which can result in vision impairment or loss, is the most frequent symptom ...
Amy D. Shapiro, Marzia Menegatti, Roberta Palla, Marco Boscarino, Christopher Roberson, Paolo Lanzi, Joel Bowen, Charles Nakar, Isaac A. Janson, Flora Peyvandi   +9 more
doaj   +1 more source

Perioperative blood loss during joint replacement: comparison between patients with and without hemophilia

Journal of Orthopaedic Surgery and Research, 2022
Background Although arthroplasty provides satisfactory pain relief, functional improvement, and reduced flexion contracture in patients with hemophilia arthropathy, bleeding remains the primary problem associated with hemophilic arthropathy joint ...
Shanyou Yuan, Lixia Song, Haoli Jiang, Jinghua Wang, Xianjia Ning, Wenxue Jiang   +5 more
doaj   +1 more source

Patient‐relevant health outcomes for hemophilia care: Development of an international standard outcomes set

Research and Practice in Thrombosis and Haemostasis, 2021
Patient‐relevant health outcomes for persons with hemophilia should be identified and prioritized to optimize and individualize care for persons with hemophilia.
E. C. van Balen, B. O’Mahony, M. Cnossen, G. Dolan, V. Blanchette, K. Fischer, D. Gue, J. O’Hara, A. Iorio, S. Jackson, B. Konkle, D. Nugent, D. Coffin, M. Skinner, C. Smit, A. Srivastava, F. V. van Eenennaam, J. G. van der Bom, S. Gouw   +18 more
semanticscholar   +1 more source

Model‐Informed Drug Development Applications and Opportunities in mRNA‐LNP Therapeutics

Clinical Pharmacology &Therapeutics, EarlyView.
The utilization of lipid nanoparticles (LNP) for encapsulating mRNA has revolutionized the field of therapeutics, enabling the rapid development of COVID‐19 vaccines and cancer vaccines. However, the clinical development of mRNA‐LNP therapeutics faces numerous challenges due to their complex mechanisms of action and limited clinical experience.
Jiawei Zhou, Rohit Rao, Monica E. Shapiro, Nessy Tania, Cody Herron, Cynthia J. Musante, Jim H. Hughes   +6 more
wiley   +1 more source

Strategies for Hemophilia Treatment, a literature review of current evidence

مجله دانشگاه علوم پزشکی بیرجند, 2023
Hemophilia is an inherited bleeding disorder caused by malfunctioning or lacking blood coagulation factor VIII (hemophilia A) or IX (hemophilia B). Currently, the main treatments for these X-linked diseases are replacement therapy using periodic and ...
Fahimeh Ghasemi, Nafiseh Erfanian, Samira Karbasi, Alireza Zomorodipour   +3 more
doaj  

Relationship between Dose, Factor IX Activity Levels and Bleeding Probability for rIX‐FP Prophylaxis in Hemophilia B: A Repeated Time‐to‐Event Analysis

Clinical Pharmacology &Therapeutics, EarlyView.
In hemophilia B, pharmacokinetic (PK)‐guided dosing of extended half‐life factor IX (EHL‐FIX) concentrates can secure targeted FIX exposure. Target FIX activity levels in plasma should be individually set primarily taking bleeding tendency into account, alongside the presence of target joints, physical activity, and preferred dosing schedules. In other
Sjoerd F. Koopman, Marjon H. Cnossen, Ron A.A. Mathot, on behalf of the OPTI‐CLOT study group and SYMPHONY consortium, S. H. Reitsma   +4 more
wiley   +1 more source

Pharmacokinetics Guided Personalized Management of Hemophilia Rehabilitation: A Chinese Consensus Based on Modified Delphi

罕见病研究
Hemophilia is a collection of hereditary bleeding disorders that are X-linked recessive. Patients often experience recurrent bleeding in joints, leading to a cascade of complications including chronic synovitis, hemophilic arthropathy, muscle atrophy ...
Hemophilia Treatment Center Collaborative Network of China
doaj   +1 more source

Optimizing Patient Registries for Regulatory Decision Making ‐ Key Learnings From an HMA/EMA Multistakeholder Workshop

Clinical Pharmacology &Therapeutics, EarlyView.
The Joint Heads of Medicines Agencies and European Medicines Agency's (HMA/EMA) big data initiative paves the way for better integration of real‐world data, including data from patient registries, into regulatory decisions on medicines. This article focuses on the outcome of a two‐day multistakeholder workshop organized by EMA in 2024, which explored ...
Kelly Plueschke, Carla Jonker, Hanna Kankanen, Thorsten Vetter, Bruno Sepodes, Lutz Naehrlich, Jan Hillert, Gracy Crane, Sabine Straus, Paolo Foggi, Simona Martin, Christina Kyriakopoulou, Peter Mol, Francesca Day, Kieran Breen, Neil Bennett, Mencia de Lemus Belmonte, Simon Bennett, Patrice Verpillat, Kit C. B. Roes, Ana Cochino, Franz Schaefer, Jesús María Hernández‐Rivas, Patricia McGettigan, Peter Arlett   +24 more
wiley   +1 more source

Population-based prevalence and mutational landscape of von Willebrand disease using large-scale genetic databases

npj Genomic Medicine, 2023
Von Willebrand disease (VWD) is a common bleeding disorder caused by mutations in the von Willebrand factor gene (VWF). The true global prevalence of VWD has not been accurately established.
Omid Seidizadeh, Andrea Cairo, Luciano Baronciani, Luca Valenti, Flora Peyvandi   +4 more
doaj   +1 more source