Results 91 to 100 of about 113,823 (359)

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

Pharmacokinetics Guided Personalized Management of Hemophilia Rehabilitation: A Chinese Consensus Based on Modified Delphi

open access: yes罕见病研究
Hemophilia is a collection of hereditary bleeding disorders that are X-linked recessive. Patients often experience recurrent bleeding in joints, leading to a cascade of complications including chronic synovitis, hemophilic arthropathy, muscle atrophy ...
Hemophilia Treatment Center Collaborative Network of China
doaj   +1 more source

Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques [PDF]

open access: yes, 2002
The feasibility, safety, and efficacy of liver-directed gene transfer was evaluated in 5 male macaques (aged 2.5 to 6.5 years) by using a recombinant adeno-associated viral (rAAV) vector (rAAV-2 CAGG-hFIX) that had previously mediated persistent ...
Davidoff, AM   +12 more
core   +1 more source

Long-term outcomes of prophylaxis with a recombinant factor VIII Fc or recombinant factor IX Fc in patients with hemophilia previously treated on demand [PDF]

open access: gold, 2023
María Román   +7 more
openalex   +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 Comparison between Recombinant Activated Factor VII (Aryoseven) and Novoseven in Patients with Congenital Factor VII Deficiency [PDF]

open access: yes, 2015
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  

Hemophilia A and B: molecular and clinical similarities and differences

open access: yesHaematologica, 2019
Several lines of evidence seem to suggest that the clinical features of hemophilia A and B patients may differ in several points. Genetic background shows a prevalence of missense mutations in hemophilia B compared to the high percentage of mutations ...
G. Castaman, Davide Matino
semanticscholar   +1 more source

Potential Biochemical Markers and Radiomorphometric Indices as Predictors of Reduced Bone Mass in Patients with Congenital Hemophilia [PDF]

open access: gold, 2022
Sylwia Czajkowska   +6 more
openalex   +1 more source

Chemical Chaperones Improve Protein Secretion and Rescue Mutant Factor VIII in Mice with Hemophilia A. [PDF]

open access: yes, 2012
nefficient intracellular protein trafficking is a critical issue in the pathogenesis of a variety of diseases and in recombinant protein production.
Abriss, Daniela   +9 more
core   +2 more sources

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

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