Results 31 to 40 of about 18,669 (233)

Haemophilia care in Iraq; a multi-centre study

Journal of the Pakistan Medical Association
Objectives: To evaluate the level of care available to haemophilia patients. Method: The descriptive, retrospective analytical study was conducted from December 15, 2020, to March 1, 2021, after approval from the Mustansiriyah University, Baghdad ...
Nidal Karim Al Rahal, Meaad Kadhum Hassan, Obeida Amir Abid, Azeezah Mohammed Mohsin   +3 more
doaj   +1 more source

A USABILITY STUDY ASSESSING HANDLING AND PREFERENCE OF THE CONCIZUMAB PEN-INJECTOR IN PATIENTS WITH HAEMOPHILIA AND CAREGIVERS

Hematology, Transfusion and Cell Therapy, 2023
Introduction: Concizumab is a once-daily prophylactic treatment in development for haemophilia A or B with or without inhibitors, and it is potentially the first treatment for haemophilia patients to be delivered subcutaneously using a prefilled, multi ...
NK Rasmussen, B Berg, JS Neergaard, S Shah, G Ter-Borch, T Sparre, GG Fabbron   +6 more
doaj   +1 more source

Engineering Biology Beyond Single Genes: Advances and Challenges in Multiplex Genome Editing

Animal Research and One Health, EarlyView.
Multiplex genome editing is transforming genome engineering from single‐gene perturbation to network‐level control, yet its broader application remains limited by challenges in gRNA array engineering, delivery technologies, and safety management. Emerging AI‐driven approaches are accelerating guide RNA design and CRISPR effector optimization for ...
Linli Wang, Yongbin Liu, Hongbing Han
wiley   +1 more source

Real-World Experience with Eptacog Beta for On-Label and Off-Label Indications: The Spanish Experience

Pharmaceuticals
Background: Eptacog beta is a novel recombinant activated factor VII (rFVIIa). Preclinical studies have shown the product has a similar profile to eptacog alfa.
Jose Manuel Martin de Bustamante, María Isabel Rivas-Pollmar, Natalia Acedo, Nora Butta, Victor Jimenez-Yuste, Maria Teresa Álvarez-Román   +5 more
doaj   +1 more source

Precision medicine in paediatrics: Progress and priorities

British Journal of Clinical Pharmacology, EarlyView.
Precision medicine is revolutionizing personalized healthcare, advancing both diagnostics and therapeutics at an unprecedented pace. Reviewing the paediatric applications of pharmacometrics, pharmacogenomics and advanced therapy medicinal products highlights not only the relevance of these exciting innovations to frontline care but also the significant
Nicola Husain, Claire Booth, Joseph F. Standing, Charlotte I. S. Barker   +3 more
wiley   +1 more source

Longitudinal Assessment of Joint Health in Paediatric Haemophilia Using the HEAD-US Ultrasound Scoring System: The ULTRA Study

Life
Background: Early detection of joint damage in haemophilia is essential to prevent haemophilic arthropathy. This study longitudinally evaluated joint health in paediatric patients with haemophilia in Greece using the Haemophilia Early Arthropathy ...
Aikaterini Michalopoulou, Olympia Papakonstantinou, Athina Dettoraki, Dimitrios Syrengelas, Vassiliki Bizimi, Konstantina Dakou, Miltiades Kyprianou, Vassiliki Spoulou, Helen Pergantou, Christina Kanaka-Gantenbein   +9 more
doaj   +1 more source

Cell and Gene Therapies Manufacturing Challenges and Integrated Good Manufacturing Practices Solutions: A Lifecycle Perspective

Biotechnology and Bioengineering, EarlyView.
Lifecycle perspective on cell and gene therapy manufacturing challenges and enabling GMP solutions. ABSTRACT Cell and gene therapies (CGTs) are revolutionizing modern medicine; however, making these advanced medicines scalable and readily available to commercial manufacturers worldwide is a major challenge. The number of approved CGT products continues
Rajath Samaga, Udisha Singh, Sachin Dattram Pawar   +2 more
wiley   +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

Human biomarker navigator

iMeta, EarlyView.
The Human Biomarker Navigator integrates the disease continuum, biomarker dynamics, cross‐organ biomarker networks, biomarker classification, and technology‐driven paradigms. It maps how biomarkers link multi‐system physiology and pathology across the nervous, respiratory, endocrine, circulatory, immune, digestive, urinary, reproductive, and ...
Meng‐Yao Li, Qian Zhang, Zijie Wang, Zeping Gui, Jing Liu, Jingda Li, Maojun Jiang, Zukang Chang, Qingqing Han, Bendong Yang, Tariq Saeed, Jun Li, Jinghan Jia, Chenghu Song, Tong Shao, Tianhuan Peng, Xueying Zhang, Pingping Guo, Jun Luo, Gangao Yang, Yuan Wan, Gokhan Zengin, Quan Yuan, Lingru Li, Xiaohui Fan, Yang Luo, Lvyun Zhu, Yanfei Zheng, Wenjun Mao, Wenlong Sun   +29 more
wiley   +1 more source

Time‐ and Temperature‐Dependent Response of Acquired FV Inhibitors: Implications for Laboratory Diagnosis and Clinical Management

Journal of Clinical Laboratory Analysis, EarlyView.
This in vitro workflow illustrates the time‐and temperature‐dependent behavior of acquired FV inhibitors. Patient plasma was centrifuged and serially diluted, followed by parallel incubation at room temperature and 37°C. Serial measurements of APTT mixing test and residual FV activity were recorded across incubation time points to monitor inhibitor ...
Lihua Zhang, Mingjie Li, Zhiqiang Xie, Junxiang Yao, Yingping Cao, Lihui Chen, Meihua Wang   +6 more
wiley   +1 more source