[HTML][HTML] Αlpha-thalassemia: A practical overview

KM Musallam, MD Cappellini, TD Coates, KHM Kuo… - Blood Reviews, 2024 - Elsevier
Abstract α-Thalassemia is an inherited blood disorder characterized by decreased synthesis
of α-globin chains that results in an imbalance of α and β globin and thus varying degrees of …

[HTML][HTML] Pharmacogenomics of Drugs Used in β-Thalassemia and Sickle-Cell Disease: From Basic Research to Clinical Applications

R Gambari, AD Waziri, H Goonasekera… - International journal of …, 2024 - mdpi.com
In this short review we have presented and discussed studies on pharmacogenomics (also
termed pharmacogenetics) of the drugs employed in the treatment of β-thalassemia or Sickle …

[HTML][HTML] Addressing Thalassaemia Management from Patients' Perspectives: An International Collaborative Assessment

EC Economidou, M Angastiniotis, D Avraam… - Medicina, 2024 - mdpi.com
Background and Objectives: The effective management of chronic diseases, particularly
hereditary and rare diseases and thalassaemia, is an important indicator of the quality of …

[HTML][HTML] Global, regional, and national burden of thalassemia, 1990–2021: a systematic analysis for the global burden of disease study 2021

Y Tuo, Y Li, Y Li, J Ma, X Yang, S Wu, J Jin, Z He - Eclinicalmedicine, 2024 - thelancet.com
Background Anemia is a significant contributor to the global disease burden, of which
thalassemia is the most common hereditary anaemic disease. Previous estimates were …

[HTML][HTML] Evaluation of Mono- and Bi-Functional GLOBE-Based Vectors for Therapy of β-Thalassemia by HBBAS3 Gene Addition and Mutation-Specific RNA …

L Koniali, C Flouri, MI Kostopoulou, NY Papaioannou… - Cells, 2023 - mdpi.com
Therapy via the gene addition of the anti-sickling βAS3-globin transgene is potentially
curative for all β-hemoglobinopathies and therefore of particular clinical and commercial …

[HTML][HTML] Insights in thalassemia: from genomics to clinical practice

CLT Silao, ZA Latiff, P Kountouris, BA Zilfalil - Frontiers in Pediatrics, 2023 - frontiersin.org
Thalassemia, a common inherited autosomal recessive disorder, is characterized by
inefficient or absent hemoglobin synthesis, resulting in various severities of anemia (1) …

[HTML][HTML] Special Issue “Personalized Medicine in Blood Disease of Children”

A Ceci, P Kountouris, A Didio, F Bonifazi - Journal of Personalized …, 2024 - mdpi.com
Personalized medicine is defined as a medical model using the characterization of
individuals' phenotypes and genotypes (eg, molecular profiling, medical imaging, and …

[HTML][HTML] Quality of Life of Children with Thalassemia in Indonesia

R Rahmah, SNN Makiyah - IJNP (Indonesian Journal of Nursing …, 2022 - journal.umy.ac.id
Background: Thalassemia is an inherited disorder of hemoglobin (Hb) synthesis. Treatment
of thalassemia has not yet reached the level of cure. Globally, the management of …

Hemoglobin β-Globin Variants In Hispanic Patients: An Institutional Experience From Dallas, Texas

M Firan, CF Timmons, JY Park, M Mitui MHA… - Hemoglobin, 2023 - Taylor & Francis
Hemoglobinopathies are the most common single-gene disorders in humans. There are
1,424 variants of human hemoglobin described with 951 involving the β-globin gene …

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S Badawy, E Severin, CLT Silao… - … : From Genomics to …, 2024 - books.google.com
Thalassemia, an autosomal recessive hematologic disease is becoming a serious health
problem worldwide with its high. prevalence and incidence (1). Thalassemia is a condition in …