Results 71 to 80 of about 12,413,959 (307)
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
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
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
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
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]
WOS ...
Kavakli, K +3 more
openaire +2 more sources
Christmas Disease (Hemophilia B) A Case Report
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
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
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
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

