Results 71 to 80 of about 12,413,959 (307)

Haemostatic action of a topical foam-based patch (VELSEAL-T) in haemophiliac patients with external bleeding

open access: yesThe Journal of Haemophilia Practice, 2020
Haemophilia is an X-linked congenital bleeding disorder due to deficiency of coagulation factor VIII (in haemophilia A) or factor IX (in haemophilia B) caused by mutations of the respective clotting factor genes.
Dutta Anupam   +3 more
doaj   +1 more source

Adaptive Machine Learning Framework for Optimizing the Affinity Purification of Adeno‐Associated Viral Vectors

open access: yesBiotechnology and Bioengineering, EarlyView.
ABSTRACT Adeno‐associated viral (AAV) vectors for gene therapy are becoming integral to modern medicine, providing therapeutic options for diseases once deemed incurable. Currently, viral vector purification is a critical bottleneck in the gene therapy industry, impacting product efficacy and safety as well as accessibility and cost to patients ...
Kelvin P. Idanwekhai   +9 more
wiley   +1 more source

Optimization of CEST MRI Reporter Protein Design Using Cation‐Pi Networks

open access: yesChemistry – A European Journal, EarlyView.
A novel engineering approach can produce reporter proteins for cell and viral therapy tracking with unique magnetic resonance imaging (MRI) signatures, detectable with chemical exchange saturation transfer (CEST). We discover how cation‐π interactions between amino acid groups can help us fine‐tune magnetic resonance properties for noninvasive ...
David E. Korenchan   +8 more
wiley   +1 more source

Cost‐effectiveness of fenfluramine as add‐on treatment in the management of Dravet Syndrome: A real‐world multicenter study

open access: yesEpilepsia Open, EarlyView.
Abstract Objective Dravet syndrome (DS) is a rare disease with a high clinical and socioeconomic impact on patients, society, and the healthcare system. The recent approval of therapies such as fenfluramine (FFA) has transformed the treatment landscape; however, data on their cost‐effectiveness are still scarce. This study evaluates the real‐world cost‐
Paolo A. Cortesi   +15 more
wiley   +1 more source

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

open access: yesHematology, 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   +6 more
doaj   +1 more source

Ddavp treatment in children with haemophilia b [PDF]

open access: yesBritish Journal of Haematology, 2001
WOS ...
Kavakli, K   +3 more
openaire   +2 more sources

Christmas Disease (Hemophilia –B) – A Case Report

open access: yesBangabandhu 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   +5 more
doaj   +1 more source

A novel knee implant for total knee arthroplasty meets expectations at 10 years. First long‐term follow‐up report of clinical outcomes and survivorship

open access: yesKnee Surgery, Sports Traumatology, Arthroscopy, EarlyView.
Abstract Purpose Achieving a “forgotten knee” after total knee arthroplasty (TKA) remains a primary goal in modern knee replacement surgery. Anatomic implant designs aim to replicate native knee anatomy and kinematics, potentially improving patient satisfaction and functional outcomes.
Alice Montagna   +6 more
wiley   +1 more source

Real‐world comparative analysis of bleeding complications and health‐related quality of life in patients with haemophilia A and haemophilia B

open access: yesHaemophilia, 2018
Clinical severity and impact of haemophilia on quality of life have been generally considered to be lower for haemophilia B (HB) compared with haemophilia A (HA) patients.
J. Booth   +6 more
semanticscholar   +1 more source

Antithrombin: Deficiency, Diversity, and the Future of Diagnostics

open access: yesMass Spectrometry Reviews, EarlyView.
ABSTRACT Our healthcare system provides reactive sick‐care, treating patients after symptoms have appeared by prescription of generic and often suboptimal therapy. This strategy brings along high costs and high pressure which is not sustainable.
Mirjam Kruijt   +2 more
wiley   +1 more source

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