Thalassemia (severe form)
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Publications and studies (1521)
- 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. A Small Key for a Heavy Door: Genetic Therapies for the Treatment of Hemoglobinopathies. (2021/02/04) ♡
- 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. Update on Clinical Ex Vivo Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases. (2021/02/03) ♡
- Development and clinical application of a preimplantation genetic testing for monogenic disease (PGT-M) for beta thalassemia in Vietnam. (2021/02/01) ♡
- Genotyping of blood groups in alloimmunized patients with β-thalassemia major by T-ARMS-PCR and multiplex-aso-pcr. (2021/02/01) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Hb Athens-Georgia (beta 40(C6) Arg > Lys, HBB:c.122G > A) with a single α-globin gene (Hb H disease) in a Thai family: molecular, hematological, and diagnostic aspects. (2021/02/01) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. A case of hyperhaemolysis syndrome in a pregnant Chinese woman with β-thalassemia during perinatal transfusion. (2021/02/01) ♡
- Bayesian spatial modeling of transfusion-dependent β-thalassemia incidence rate in Fars Province, Southern Iran. (2021/02/01) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Global loss of Tfr2 with concomitant induced iron deficiency greatly ameliorates the phenotype of a murine thalassemia intermedia model. (2021/02/01) ♡
- Identification of Circulating Endocan-1 and Ether Phospholipids as Biomarkers for Complications in Thalassemia Patients. (2021/01/26) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia. (2021/01/21) ♡
- Consequences of β-Thalassemia or Sickle Cell Disease for Ovarian Follicle Number and Morphology in Girls Who Had Ovarian Tissue Cryopreserved. (2021/01/15) ♡
- Induction of therapeutic levels of HbF in genome-edited primary β(0) 39-thalassaemia haematopoietic stem and progenitor cells. (2021/01/01) ♡
- 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. Genetic epidemiology of hemoglobinopathies among Iraqi Kurds. (2021/01/01) ♡
- Marrow Fat Content and Composition in β-Thalassemia: A Study using (1) H-MRS. (2021/01/01) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Tolerance induction to deferasirox in a child with transfusion-dependent beta thalassemia. (2021/01/01) ♡
- 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. Hemoglobinopathy and pediatrics in the time of COVID-19. (2021/01/01) ♡
- Systematic reviewiAll research on one question is searched according to fixed rules and compared side by side, so that no studies with poor outcomes are missed. The spectrum of beta-thalassemia mutations in the 22 Arab countries: a systematic review. (2021/01/01) ♡
- Systematic reviewiAll research on one question is searched according to fixed rules and compared side by side, so that no studies with poor outcomes are missed. β-Thalassemia Major and Coronavirus-19, Mortality and Morbidity: a Systematic Review Study. (2021/01/01) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Establishment and characterization of induced pluripotent stem cell line (IGIBi002-A) from a β-thalassemia patient with IVS1-5 mutation by non-integrating reprogramming approach. (2021/01/01) ♡
- Red cell alloantibodies in beta-thalassaemia major patients' blood referring to the regional blood transfusion center of Tehran, Iran. (2021/01/01) ♡
- Malnutrition, Its Attributes, and Impact on Quality of Life: An Epidemiological Study among β-Thalassemia Major Children. (2021/01/01) ♡
- Evaluation of iron overload by cardiac and liver T2* in β-thalassemia: Correlation with serum ferritin, heart function and liver enzymes. (2021/01/01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Genotypic and Clinical Analysis of a Thalassemia Major Cohort: An Observational Study. (2021/01/01) ♡
- 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. Prevention programmes and prenatal diagnosis for beta thalassemia in Pakistan: A narrative review. (2021/01/01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Clinical Experience of Thalidomide in Thalassemic Patients (2021-11-24) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (THALA-RAP) (2021-11-12) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Impact of the Preparation Method of Red Cell Concentrates on Transfusion Indices in Thalassemic Patients (2021-11-12) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Assessment of Cognitive Functions, Psychological Symptoms, and Quality of Life Among B Thalassemia Patients and Their Clinical Correlates. (2021-11-03) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Phase 2a Pilot Study of NBMI Treatment in Patients With Beta Thalassemia Major, Requiring Iron Chelation (2021-08-26) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity (2021-08-24) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Cord Blood Transfusion In Preterm Neonates (CB-TrIP) (2021-08-17) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Adherence to HU and HRQOL in Patients With Sickle Cell Disease: An Intervention Study Using HU-Go App (2021-07-29) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia (2021-07-29) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Iron Status in BTM With Blood Transfusion (2021-07-19) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia (2021-07-16) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety Study for Beta Thalassemia Subjects on PTG-300 (2021-07-16) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Thrombin Generation in Beta-thalassemia Major (2021-07-15) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Evaluation of Heart Status in Patients of Beta Thalassemia Using Echocardiogram (2021-06-18) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload (2021-06-14) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment (2021-06-14) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Genetic Variants Affecting the Clinical Severity of Beta Thalassemia (2021-06-11) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Association of Hb F Level With Clinical Severity of Beta Thalassemia (2021-06-11) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload (2021-06-10) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Treatment of Iron Overload Requiring Chelation Therapy (2021-06-02) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. PK Study of Ticagrelor in Children Aged Less Than 24 Months, With Sickle Cell Disease (HESTIA4) (2021-05-10) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients (2021-05-04) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Observing the Changes of Endocrine and Metabolism in Patients With Thalassemia Major (2021-02-18) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Adiponectin, IL-6 and hsC-RP in Relation to Carotid Intima-media Thickness in B-thalassemia Patients (2021-02-16) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. The Role of OCTA in Patients Affected by Beta Thalassemia (2021-02-03) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Hydroxy Urea, Omega 3, Nigella Sativa, Honey on Oxidative Stress and Iron Chelation in Pediatric Major Thalassemia (2021-01-27) ♡
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