Results 61 to 70 of about 5,832,501 (223)

IMPROVED SURVIVAL IN BETA THALASSEMIA MAJOR [PDF]

Pediatric Research, 1987
The natural history of transfusion (tx) dependent Beta thalassemia (thai) is changing owing to recent advances and progress in its clinical management. Historically, thal pts developed hemochromatosis and succumbed in the second decade of life with myocardial disease.
P J Giardina, K Ehlers, M Lesser, M Connelly, D Weinstein, E Jew, M W Hilgartner   +6 more
openaire   +1 more source

In Utero HSC Transplantation for Sickle Cell Disease: A Potential Therapeutic Approach That Overcomes Complications of Current Therapies

Prenatal Diagnosis, EarlyView.
ABSTRACT Sickle cell disease (SCD) affects millions worldwide but has limited treatment options, most of which carry significant side effects. At present, the only curative treatment for SCD is allogeneic or gene‐modified autologous hematopoietic stem cell (HSC) transplantation (Tx).
Oluwaseun O. Babatunde, Madeleine G. Bibby, Anthony Atala, Graça Almeida‐Porada, Christopher D. Porada   +4 more
wiley   +1 more source

The Correlation between Ferritin Levels and Glutation (GSH) Levels in Major Beta Thalassemia Patient at dr. Soebandi Jember Hospital

Journal of Agromedicine and Medical Sciences (AMS), 2018
Repeated blood transfusions in patient with Major Beta Thalassemia leads to iron overload marks by the increased of serum ferritin levels. Free iron caused the change of H2O2 to oxidants by fenton reaction and caused oxidative stress.
Amalia Nur Zahra, Rini Riyanti, Elly Nurus Sakinah   +2 more
doaj   +1 more source

Non‐Invasive Prenatal Diagnosis of Haemoglobinopathies: From Concept to Clinical Reality

Prenatal Diagnosis, EarlyView.
ABSTRACT Haemoglobinopathies are the most prevalent monogenic disorders worldwide, contributing significantly to childhood morbidity and mortality. Preventive strategies, including carrier screening and prenatal diagnosis, have successfully reduced disease incidence in regions with established programmes.
Irene Leonidou, Leila Sallie, Morgan Jean Bell‐Ricketts, Alexandros Abdel Massih, Lisa Joy Kaiser, Ashley Hoi Kiu Wong, María Teresa Martín Monreal, Panicos Shangaris   +7 more
wiley   +1 more source

Beta Thalassemia: An Overview of Epidemiology, Clinical Features, Diagnosis, and Management [PDF]

پزشکی بالینی ابن سینا
Background and Objective: Thalassemia is an inherited blood disorder with an autosomal recessive inheritance pattern caused by the reduced or absent production of globin chains.
Ghasem Miri-Aliabad
doaj  

Association Between Plasma Cystatin C Concentration and Urine Osmolality in Adults with Different Forms of Beta-Thalassemia: A Cross-Sectional Study in Vietnam

Thalassemia Reports
Objective: To determine plasma cystatin C concentrations, urine osmolality and their relationship with disease severity in beta-thalassemia patients. Methods: A cross-sectional study was conducted on 234 patients with beta-thalassemia, including equal ...
Loan Do Thi Thanh, Anh Nguyen Ngoc, Kien Nguyen Trung, Ha Nguyen Thi Thu, Dung Nguyen Huu, Thang Le Viet   +5 more
doaj   +1 more source

Gene Editing for Haemophilia—The Next Frontier

Haemophilia, EarlyView.
ABSTRACT The recently approved haemophilia A and B gene therapies via adeno‐associated virus (AAV) showed a promising therapeutic response after a single injection, but there are still limitations, including the potential loss of transgene expression and restriction in adults.
Mirko Pinotti, Gregory A. Newby, Sundar Selvaraj, Steven W. Pipe   +3 more
wiley   +1 more source

CRISPR Gene Therapy: A Promising One-Time Therapeutic Approach for Transfusion-Dependent β-Thalassemia—CRISPR-Cas9 Gene Editing for β-Thalassemia

, 2023
β-Thalassemia is an inherited hematological disorder that results from genetic changes in the β-globin gene, leading to the reduced or absent synthesis of β-globin.
Gayathri N. Silva, Kesari Warnakulasuriya, Udani Gamage, Sonali Hansika   +3 more
core   +1 more source

Lack of awareness about thalassemia; a social factor in the propagation of beta thalassemia gene in Pakistan: A population study

Mediterranean Journal of Hematology and Infectious Diseases, 2014
Background: Beta thalassemia is the most common genetic disorder in Pakistan. Thalassemia is a preventive disease a fact well demonstrated by countries such as Italy, Greece and Cyprus.
Muhammad Usman
doaj  

Adrenal Insufficiency in Patients with Beta-Thalassemia Major in the Southeast of Iran

International Journal of Hematology-Oncology and Stem Cell Research, 2022
Beta-thalassemia is a hereditary blood disorder in which the synthesis of the hemoglobin beta chain is halted or severely diminished, chiefly due to genetic mutations.
Ghasem Miri-Aliabad, Maryam Nakhaie Moghadam, Majid Naderi, Mahdie Saravani, Ramin Saravani, Saman Sargazi, Milad Shirvaliloo   +6 more
doaj