Transforming Hemophilia A Care: Insights into New Therapeutic Options

Hemophilia A is a hereditary bleeding disorder characterized by a deficiency in clotting factor VIII, leading to significant morbidity and a reduced quality of life. This review provides an updated overview of the current understanding of hemophilia A, highlighting its genetic underpinnings and adva...

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Main Authors: Iasmina-Maria Iurea, Emilia Severin, Alexandra Matei
Format: Article
Language:English
Published: MDPI AG 2024-11-01
Series:Life
Subjects:
Online Access:https://www.mdpi.com/2075-1729/14/12/1568
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author Iasmina-Maria Iurea
Emilia Severin
Alexandra Matei
author_facet Iasmina-Maria Iurea
Emilia Severin
Alexandra Matei
author_sort Iasmina-Maria Iurea
collection DOAJ
description Hemophilia A is a hereditary bleeding disorder characterized by a deficiency in clotting factor VIII, leading to significant morbidity and a reduced quality of life. This review provides an updated overview of the current understanding of hemophilia A, highlighting its genetic underpinnings and advancements in treatment strategies. A literature review was conducted using various available databases. Relevant studies on hemophilia A, covering genetics and treatment options, were selected and summarized. Recent developments in gene therapy are discussed, showcasing their potential to offer long-term solutions and reduce the burden of treatment. Additionally, the review addresses global disparities in care and policy implications, emphasizing the need for comprehensive healthcare frameworks to improve outcomes for individuals living with hemophilia A worldwide. By synthesizing recent findings and insights, this review aims to inform clinicians and policymakers about the evolving landscape of hemophilia A management and the necessity for equitable access to care.
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spelling doaj-art-cb01626b95ab45aba1230a3f3354cbc72024-12-27T14:35:56ZengMDPI AGLife2075-17292024-11-011412156810.3390/life14121568Transforming Hemophilia A Care: Insights into New Therapeutic OptionsIasmina-Maria Iurea0Emilia Severin1Alexandra Matei2Genetics Department, Carol Davila University of Medicine and Pharmacy, Dionisie Lupu 37 Street, 020021 Bucharest, RomaniaGenetics Department, Carol Davila University of Medicine and Pharmacy, Dionisie Lupu 37 Street, 020021 Bucharest, RomaniaGenetics Department, Carol Davila University of Medicine and Pharmacy, Dionisie Lupu 37 Street, 020021 Bucharest, RomaniaHemophilia A is a hereditary bleeding disorder characterized by a deficiency in clotting factor VIII, leading to significant morbidity and a reduced quality of life. This review provides an updated overview of the current understanding of hemophilia A, highlighting its genetic underpinnings and advancements in treatment strategies. A literature review was conducted using various available databases. Relevant studies on hemophilia A, covering genetics and treatment options, were selected and summarized. Recent developments in gene therapy are discussed, showcasing their potential to offer long-term solutions and reduce the burden of treatment. Additionally, the review addresses global disparities in care and policy implications, emphasizing the need for comprehensive healthcare frameworks to improve outcomes for individuals living with hemophilia A worldwide. By synthesizing recent findings and insights, this review aims to inform clinicians and policymakers about the evolving landscape of hemophilia A management and the necessity for equitable access to care.https://www.mdpi.com/2075-1729/14/12/1568hemophilia Ableeding disorderhemophilia geneticsgene therapyquality of life
spellingShingle Iasmina-Maria Iurea
Emilia Severin
Alexandra Matei
Transforming Hemophilia A Care: Insights into New Therapeutic Options
Life
hemophilia A
bleeding disorder
hemophilia genetics
gene therapy
quality of life
title Transforming Hemophilia A Care: Insights into New Therapeutic Options
title_full Transforming Hemophilia A Care: Insights into New Therapeutic Options
title_fullStr Transforming Hemophilia A Care: Insights into New Therapeutic Options
title_full_unstemmed Transforming Hemophilia A Care: Insights into New Therapeutic Options
title_short Transforming Hemophilia A Care: Insights into New Therapeutic Options
title_sort transforming hemophilia a care insights into new therapeutic options
topic hemophilia A
bleeding disorder
hemophilia genetics
gene therapy
quality of life
url https://www.mdpi.com/2075-1729/14/12/1568
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