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Blood and bone marrow
Thalassemia (severe form)
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Publications and studies (1521)
- Association of FOXO3 polymorphism (rs3800231) and clinical subphenotypes of beta thalassemic individuals. (2022/04/01) ♡
- Beta thalassemia major: the invaluable role of electrocardiography and echocardiography. (2022/04/01) ♡
- Incidental Detection of Hemoglobin Variants During Evaluation of HbA1c. (2022/04/01) ♡
- Plasma proteome profiling combined with clinical and genetic features reveals the pathophysiological characteristics of β-thalassemia. (2022/03/16) ♡
- Structural switching/polymorphism by sequential base substitution at quasi-palindromic SNP site (G → A) in LCR of human β-globin gene cluster. (2022/03/15) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. A novel SUPT5H variant associated with a beta-thalassaemia trait. (2022/03/01) ♡
- Curative therapy for hemoglobinopathies: an International Society for Cell & Gene Therapy Stem Cell Engineering Committee review comparing outcomes, accessibility and cost of ex vivo stem cell gene therapy versus allogeneic hematopoietic stem cell transplantation. (2022/03/01) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Case study of betibeglogene autotemcel gene therapy in an adult Greek patient with transfusion-dependent β-thalassaemia of a severe genotype. (2022/03/01) ♡
- A New Mutation, Hb A(2)-Canakkale [δ10(A7)Ala→Val; HBD: c.32C>T], and Other Well-Known δ Variants Identified in a Selected Cohort with Low Hb A(2) Levels. (2022/03/01) ♡
- Newborn Screening for Sickle Cell Disease Among Tribal Populations in the States of Gujarat and Madhya Pradesh in India: Evaluation and Outcome Over 6 Years. (2022/02/15) ♡
- Phase 3 studyiResearch in a large group of patients, the final step before a treatment can be approved. What comes out here carries significant weight. Betibeglogene Autotemcel Gene Therapy for Non-β(0)/β(0) Genotype β-Thalassemia. (2022/02/03) ♡
- Extended family thalassemia screening as a feasible alternative method to be implemented in identifying carriers in West Java, Indonesia. (2022/02/01) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Can ruxolitinib avert splenectomy in patients with thalassaemia: a short term case series. (2022/02/01) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Cell erythroid maturation approach: a new paradigm in the road map towards a cure for β-thalassaemia syndromes. (2022/02/01) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Impaired bone marrow microenvironment and stem cells in transfusion-dependent beta-thalassemia. (2022/02/01) ♡
- Genetic research and clinical analysis of β-globin gene cluster deletions in the Chinese population of Fujian province: A 14-year single-center experience. (2022/02/01) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Risk of mortality from anemia and iron overload in nontransfusion-dependent β-thalassemia. (2022/02/01) ♡
- Improved Non-Invasive Preimplantation Genetic Testing for Beta-Thalassemia Using Spent Embryo Culture Medium Containing Blastocoelic Fluid. (2022/01/20) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. New Frontiers: Precise Editing of Allergen Genes Using CRISPR. (2022/01/17) ♡
- An online alpha-thalassemia carrier discrimination model based on random forest and red blood cell parameters for low HbA(2) cases. (2022/01/15) ♡
- Dynamics of Individual Red Blood Cells Under Shear Flow: A Way to Discriminate Deformability Alterations. (2022/01/05) ♡
- Subclinical nephrotoxicity in patients with beta-thalassemia: role of urinary kidney injury molecule. (2022/01/01) ♡
- Survival and complications in patients with haemoglobin E thalassaemia in Sri Lanka: a prospective, longitudinal cohort study. (2022/01/01) ♡
- Impact of HFE-2 and HAMP Gene Variations on Iron Overload in Pediatric Patients with Non-Transfusion Dependent Thalassemia: A Pilot Study. (2022/01/01) ♡
- Endothelial Activation Markers in Polytransfused Children with Beta Thalassemia: Study from a Tertiary Care Centre in India. (2022/01/01) ♡
- Iron parameters in pregnant women with beta-thalassaemia minor combined with iron deficiency anaemia compared to pregnant women with iron deficiency anaemia alone demonstrate the safety of iron supplementation in beta-thalassaemia minor during pregnancy. (2022/01/01) ♡
- Primary HBB gene mutation severity and long-term outcomes in a global cohort of β-thalassaemia. (2022/01/01) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Prevalence and Management of β-Thalassemia in India. (2022/01/01) ♡
- [Application of genome editing technology in gene therapy]. (2022/01/01) ♡
- Noninvasive Prenatal Diagnosis of Beta-Thalassemia Disease by Using Digital PCR Analysis of Cell-Free Fetal DNA in Maternal Plasma. (2022/01/01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia Study (TIGET-BTHAL) (2022-11-23) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia (2022-11-10) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Clinical Study to Evaluate the Possible Efficacy and Safety of L- Carnitine and Sildenafil in Children Having Beta Thalassemia With Increased Tricuspid Regurgitant Jet Velocity (2022-10-18) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. β-globin Restored Autologous HSC in β-thalassemia Major Patients (2022-10-13) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Chelation Therapy of Iron Overload With Pyridoxal Isonicotinoyl Hydrazone (2022-09-21) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Assessment of Nutritional Status and Role of Insulin-like Growth Factor-1 in Children (2022-09-07) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Apotransferrin in Patients With β-thalassemia (2022-08-15) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Prevalence of Pulmonary Hypertension (PAH) in Patients With Thalassemia (2022-08-04) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. The Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-world Study (2022-07-18) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. The Prevelence of IVS 1-6 (T-C) [HBB:c.92 +6 T-C] Gene Mutation in Suspected Cases of β Thalassemia in Assiut University Hospitals (2022-05-11) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease (2022-04-26) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Study on the Effect of Different Risk Factors on the Growth Parameters of Thalassemic Patients in Assiut (AUCH) (2022-03-31) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Pilot Study to Assess the Safety, PK and Iron Chelating Activity of DST-0509 (Deferasirox) in Thalassemia Patients Refractory to Chelation (2022-02-01) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Shared molecular basis, diagnosis, and co-inheritance of alpha and beta thalassemia. (2021/12/31) ♡
- Native cardiac magnetic resonance T1 mapping and cardiac mechanics as assessed by speckle tracking echocardiography in patients with beta-thalassaemia major. (2021/12/31) ♡
- Correlation between Serum Fatty Acid Binding Protein 4 (FABP4) Levels and Cardiac Function in Patients with Thalassemia Major. (2021/12/27) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Hearing Loss in Beta-Thalassemia: Systematic Review. (2021/12/25) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Mitapivat, a novel pyruvate kinase activator, for the treatment of hereditary hemolytic anemias. (2021/12/21) ♡
- Quality of Life and Burden of Disease in Italian Patients with Transfusion-Dependent Beta-Thalassemia. (2021/12/21) ♡
- The Influence of Cardiovascular Risk Factors and Hypogonadism on Cardiac Outcomes in an Aging Population of Beta-Thalassemia Patients. (2021/12/21) ♡
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