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
Haemophilia A: pharmacoeconomic review of prophylaxis treatment versus on-demand [PDF]
Objectives. Haemophilia A is a congenital disorder of coagulation that mainly affects males and causes a considerable use of resources, especially when hemophilic patients are treated with prophylaxis.
BOCCIA, Antonio+4 more
core +2 more sources
Exploring the Potential and Advancements of Circular RNA Therapeutics
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+6 more
wiley +1 more source
A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A.
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+46 more
semanticscholar +1 more source
Performance of AAV8 vectors expressing human factor IX from a hepatic-selective promoter following intravenous injection into rats [PDF]
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+4 more
core +2 more sources
TREATMENT OF HEMOPHILIA WITH HUMAN FACTORIX PRODUCED IN MAMIMARY TISSUE OF TRANSGENIC MAMMALS [PDF]
Recombinant Factor IX characterized by a high percentage of active protein can be obtained in the milk of transgenic animals that incorporate chimeric DNA molecules according to the present invention.
Drohan, William N.+4 more
core +1 more source
Investigating the role of SARM1 in central nervous system
Sterile‐α and Toll/interleukin 1 receptor (TIR) motif‐containing protein 1 (SARM1) is a pivotal molecule that has garnered extensive attention in neuroscience. As an intracellular molecule, SARM1 possesses various crucial biological functions in the nervous system.
Junjie Wang+4 more
wiley +1 more source
Factor VIII-Mimetic Function of Humanized Bispecific Antibody in Hemophilia A.
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+9 more
semanticscholar +1 more source
A Comparison between Recombinant Activated Factor VII (Aryoseven) and Novoseven in Patients with Congenital Factor VII Deficiency [PDF]
In order to establish the efficacy and biosimilar nature of AryoSeven to NovoSeven in the treatment of congenital factor VII (FVII) deficiency, patients received either agent at 30 1/4g/kg, intravenously per week for 4 weeks, in a randomized fashion. The
Abolghasemi, H.+16 more
core
Evaluation of coagulation disorders and iron deficiency in women with heavy menstrual bleeding
Abstract Objective To explore the incidence of congenital bleeding disorders (CBD), which may result in anemia, in a large cohort of women referred for heavy menstrual bleeding (HMB) in a specialized gynecologic unit. Methods Between January 2022 and January 2024, all women referred for HMB were screened.
Lucia Rugeri+8 more
wiley +1 more source