Results 91 to 100 of about 22,643,381 (355)

Perfil clínico e epidemiológico de crianças e adolescentes com hemofilia em um centro de referência

Residência Pediátrica, 2023
OBJECTIVE: To identify clinical and epidemiological data of children and adolescents with hemophilia followed at the Center for Hematology and Hemotherapy of Piaui, characterizing them in terms of age, place of residence, type, severity, main ...
Marcela Bezerra Marques, Maria Aline Ferreira de Cerqueira   +1 more
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

INDUCTION OF TOLERANCE BY ORAL ADMINISTRATION OF FACTOR VIII AND TREATMENT OF HEMOPHILA [PDF]

, 2011
Disclosed herein is a simple method for the treatment of antigen-deficiency diseases, by orally administering to a Subject a therapeutically effective amount of the deficient anti gen, wherein the antigen is not present in a liposome.
Alpan, Oral, Kamala, Tirumalai, Matzinger, Polly, Velander, William Hugold   +3 more
core   +1 more source

Performance of AAV8 vectors expressing human factor IX from a hepatic-selective promoter following intravenous injection into rats [PDF]

, 2008
Background: Vectors based on adeno-associated virus-8 (AAV8) have shown efficiency and efficacy for liver-directed gene therapy protocols following intravascular injection, particularly in relation to haemophilia gene therapy. AAV8 has also been proposed
Baker, AH, Graham, T, McIntosh, J, Nathwani, A, Work, LM   +4 more
core   +2 more sources

Quantifying Critical Quality Attributes of Protein Therapeutics by Sodium Dodecyl Sulfate–Capillary Gel Electrophoresis With Native Fluorescence Detection

ELECTROPHORESIS, EarlyView.
ABSTRACT In the biopharmaceutical industry, the sodium dodecyl sulfate–capillary gel electrophoresis (SDS–CGE) assay is often used to assess therapeutic critical quality attributes (CQAs). Traditional detection methods for SDS–CGE methods, such as ultraviolet (UV) absorbance and laser‐induced fluorescence (LIF), are widely used but come with ...
Zaifang Zhu
wiley   +1 more source

A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A.

New England Journal of Medicine, 2016
BACKGROUND The development of neutralizing anti-factor VIII alloantibodies (inhibitors) in patients with severe hemophilia A may depend on the concentrate used for replacement therapy.
F. Peyvandi, P. Mannucci, I. Garagiola, A. El‐Beshlawy, M. Elalfy, V. Ramanan, P. Eshghi, S. Hanagavadi, R. Varadarajan, M. Karimi, M. Manglani, C. Ross, G. Young, T. Seth, Shashikant Apte, D. Nayak, E. Santagostino, M. Mancuso, Adriana C. Sandoval Gonzalez, J. Mahlangu, Santiago Bonanad Boix, M. Cerqueira, N. Ewing, C. Male, T. Owaidah, Veronica Soto Arellano, N. Kobrinsky, S. Majumdar, R. Perez Garrido, A. Sachdeva, M. Simpson, Mathew Thomas, E. Zanon, B. Antmen, K. Kavaklı, M. Manco‐Johnson, Monica Martinez, E. Marzouka, M. Mazzucconi, D. Neme, Ángeles Palomo Bravo, R. Paredes Aguilera, A. Prezotti, K. Schmitt, B. Wicklund, B. Zulfikar, F. Rosendaal   +46 more
semanticscholar   +1 more source

Exploring the Potential and Advancements of Circular RNA Therapeutics

Exploration, EarlyView.
Given the remarkable advantages in terms of stability, sustained expression profile, safety, wide range of druggable targets, scalable and cost‐effective manufacturing capabilities, circRNA is currently undergoing intensive investigation for various therapeutic applications such as vaccines, protein replacement, genetic disease treatment, gene therapy,
Lei Wang, Lianqing Wang, Chunbo Dong, Jialong Liu, Guoxin Cui, Shan Gao, Zhida Liu   +6 more
wiley   +1 more source

Surgical and Medical Co‐Management in an 82‐Year‐Old Patient With Hemophilia a Undergoing Pancreaticoduodenectomy

Health Care Science, EarlyView.
The surgical and medical co‐management (SMC) team integrates internists and surgeons to improve perioperative planning to minimize complications and promote recovery. SMCs team implemented various perioperative optimization strategies for the patient to prevent complications and enhance satisfaction.
Wenning Lu, Chaoyang Liu, Jing He, Rui Cheng   +3 more
wiley   +1 more source

Utilization evaluation of factor concentration and frequencyof bleeds among patients with haemophilia "A" and haemophilia "B" in northwest Iran [PDF]

Journal of Analytical Research in Clinical Medicine, 2013
Background Haemophilia A and B are X-linked bleeding disorders which result in decreased blood levels of coagulants. According to some studies, Hemophilia Severity Score (HSS) is higher in severe Haemophilia A(HA) than in severe Haemophilia B(HB ...
Roya Dolatkhah, Morteza Ghojazadeh, Iraj Asvadi Kermani, Zohreh Sanaat, Nasrin Tavassoli, Mehri Gholchin   +5 more
doaj   +1 more source

Factor VIII-Mimetic Function of Humanized Bispecific Antibody in Hemophilia A.

New England Journal of Medicine, 2016
BACKGROUND In patients with severe hemophilia A, standard treatment is regular prophylactic and episodic intravenous infusions of factor VIII. However, these treatments are burdensome, especially for children, and may lead to the formation of anti-factor
M. Shima, H. Hanabusa, M. Taki, T. Matsushita, Tetsuji Sato, K. Fukutake, Naoki Fukazawa, K. Yoneyama, Hiroki Yoshida, K. Nogami   +9 more
semanticscholar   +1 more source