Results 31 to 40 of about 49,324 (252)

Haemophilia A: pharmacoeconomic review of prophylaxis treatment versus on-demand [PDF]

open access: yes, 2015
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

Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy

open access: yesMolecular Therapy: Methods & Clinical Development, 2019
Hemophilia A (HA) and hemophilia B (HB) are X-linked bleeding disorders due to inheritable deficiencies in either coagulation factor VIII (FVIII) or factor IX (FIX), respectively.
Benjamin J. Samelson-Jones   +1 more
doaj   +1 more source

Von Willebrand Disease [PDF]

open access: yes, 2017
Von Willebrand Disease (VWD) is a blood clotting disorder characterized by low levels of the Von Willebrand Factor (VWF) in the blood. VWF is functions to bind platelets and promote clotting of the blood when vascular injury occurs.
Misla David, Ramon
core   +2 more sources

Preclinical evaluation of a next-generation, subcutaneously administered, coagulation factor IX variant, dalcinonacog alfa.

open access: yesPLoS ONE, 2020
IntroductionThe rapid clearance of factor IX necessitates frequent intravenous administrations to achieve effective prophylaxis for patients with hemophilia B.
Timothy C Nichols   +4 more
doaj   +1 more source

Cardiac surgery in patients with Hemophilia:is it safe?

open access: yesJournal of Cardiothoracic Surgery, 2020
Background The life expectancy of hemophiliacs is similar to that of the general population. As a result, the prevalence of age-related cardiovascular diseases has increased.
Amjad Shalabi   +9 more
doaj   +1 more source

A Case of Heel Reconstruction with a Reverse Sural Artery Flap in a Hemophilia B Patient [PDF]

open access: yesArchives of Plastic Surgery, 2012
Hemophilia B is a rare blood coagulation disorder. Complications such as bleeding and hematoma can cause necrosis of flaps, wound disruption, and the disturbance of wound healing.
Byung Kwon Lee, Jeong Su Shim
doaj   +2 more sources

In Utero Transplantation of Placenta-Derived Mesenchymal Stromal Cells for Potential Fetal Treatment of Hemophilia A. [PDF]

open access: yes, 2018
Hemophilia A (HA) is an X-linked recessive disorder caused by mutations in the factor VIII ( FVIII) gene leading to deficient blood coagulation. The current standard of care is frequent infusions of plasma-derived FVIII or recombinant B-domain-deleted ...
Farmer, Diana   +6 more
core   +1 more source

Dual‐Functional Polyphosphoesters for Gene Delivery: Synergistic Effects of Guanidinium and Hydrophobic Side Chains in Degradable Polymers

open access: yesAdvanced Healthcare Materials, EarlyView.
This study presents guanidinium‐ and indole‐functionalized polyphosphoesters as degradable, non‐viral gene delivery vectors. Through precise tuning of charge density and hydrophobicity, these polymers form stable polyplexes with low toxicity. Remarkably, minor structural changes yield up to 200‐fold differences in transfection efficiency, highlighting ...
Markus Kötzsche   +8 more
wiley   +1 more source

Recurrent bleeding after rubber band ligation diagnosed as mild hemophilia B: a case report and literature review

open access: yesBMC Surgery, 2022
Background Hemophilia is a recessive hemorrhagic disease relevant to X chromosome. In mild hemophilia cases, spontaneous bleeding is rare and the blood clotting function is normal, but severe bleeding may occur after trauma or surgery.
Xiaoying Jiang   +4 more
doaj   +1 more source

PRODUCTION OF HIGH LEVELS OF TRANSGENC FACTOR IX WITHOUT GENE RESCUE, AND ITS THERAPEUTIC USES [PDF]

open access: yes, 2011
A non-human transgenic mammalian animal, as described above, contains an exogenous double stranded DNA sequence stably integrated into the genome of the animal, which comprises cis-acting regulatory units operably linked to a DNA sequence encoding human ...
Cooper, Julian D.   +2 more
core   +1 more source

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