Results 51 to 60 of about 13,284,040 (264)
Precision medicine in paediatrics: Progress and priorities
British Journal of Clinical Pharmacology, EarlyView.Precision medicine is revolutionizing personalized healthcare, advancing both diagnostics and therapeutics at an unprecedented pace. Reviewing the paediatric applications of pharmacometrics, pharmacogenomics and advanced therapy medicinal products highlights not only the relevance of these exciting innovations to frontline care but also the significant
Nicola Husain +3 more
wiley +1 more source
Hematology ReportsBackground/Objectives: Hemophilia A is an X-linked recessive illness produced by a deficiency of coagulation factor VIII. This study aimed to evaluate serum vitamin D in hemophilic pediatric patients and its correlation with joint health and quality of ...
Aida M. S. Salem +6 more
doaj +1 more source
Cardiac surgery in patients with Hemophilia:is it safe?
Journal 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
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Immune tolerance induction in a severe hemophilia B child with low titer inhibitors
Journal of Applied Hematology, 2022 Hemophilia B is an X-linked inherited bleeding disorder caused by either the absence or reduced biosynthesis of clotting factor IX (FIX). This disorder affects approximately 1 in 30,000 male individuals worldwide. Patients with severe form (FIX
Asia Almulla, Najam Awan, Faisl Khanani
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Development of a Clinical Candidate AAV3 Vector for Gene Therapy of Hemophilia B
Human Gene Therapy, 2020 Although recombinant adeno-associated virus serotype 8 (AAV8) and serotype 5 (AAV5) vectors have shown efficacy in Phase 1 clinical trials for gene therapy of hemophilia B, it has become increasingly clear that these serotypes are not optimal for ...
Harrison C. Brown +7 more
semanticscholar +1 more source
Harnessing blood clot as a native scaffold for orchestrating tissue repairs and regeneration
BMEMat, EarlyView.The blood clot, owing to its dynamic composition and unique microenvironment, holds significant yet underappreciated potential for tissue engineering. This review systematically summarizes the pathophysiology of clot formation, the key regulatory factors shaping its microenvironment, and its applications in both pre‐clinical and clinical settings ...
Gao‐peng Dang +13 more
wiley +1 more source
PLoS ONE, 2023 IntroductionHemophilia A and B are disorders associated with the deficit of coagulation factors VIII and IX.ObjectiveWas to determine the incidence of complications in a cohort of patients diagnosed with moderate and severe hemophilia A or B under ...
Jorge E Machado Alba +9 more
doaj +1 more source
Status and future of recombinant adeno‐associated virus vector manufacturing
Biotechnology Progress, EarlyView.Abstract Sixty years of adeno‐associated virus (AAV) research illustrates a trajectory marked by basic science exploration, iterative innovation, persistent challenges, a number of clinical setbacks, as well as commercial therapeutic triumphs. This continual evolution has led to recombinant AAV (rAAV) becoming a cornerstone of modern gene therapy ...
Frank Agbogbo, David Dismuke
wiley +1 more source
PLoS 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
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Clinical Pharmacology &Therapeutics, EarlyView.Precision medicine offers the opportunity to improve the benefit–risk profile of new therapies by prospectively identifying patients most likely to respond or least likely to experience harm; however, its systematic integration into drug development remains inconsistent outside oncology.
Ingrid Holst‐Laubjerg +1 more
wiley +1 more source