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Thalassemia (severe form)
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Publications and studies (1521)
- Diagnostic Utility of High-Performance Liquid Chromatography and Its Correlation With Hematological Indices in the Evaluation of Hemoglobinopathies. (2026/05/19) ♡
- Factors associated with anemia among adolescent girls in Western India: insights from a multi-centric cross-sectional study. (2026/05/18) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Missed Wrist Septic Arthritis: A Cautionary Case Report in a Postpartum Patient With Complement Deficiency. (2026/05/17) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Compound Heterozygous Sickle Cell-Beta Thalassemia Presenting As Chronic Hemolytic Anemia With Microcytosis and Prominent Left Ventricular Trabeculation: A Case Report. (2026/05/13) ♡
- Acute erythroblastopenia due to Parvovirus B19 in hemoglobinopathies: a retrospective case series at Ibn Sina Hospital, Rabat, Morocco. (2026/05/06) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Takotsubo syndrome with concomitant coronary angioplasty and implantable cardioverter-defibrillator implantation: a case report. (2026/05/05) ♡
- [Application of the failure mode and effects analysis (FMEA) to the pharmaceutical process of Casgevy®, an ex vivo gene therapy medicinal product for beta-thalassemia]. (2026/05/01) ♡
- Ocular Abnormalities in Children with Beta Thalassemia Major at a Tertiary Care Center. (2026/05/01) ♡
- Transfusion Modalities in Thalassemia Patients: Prophylactic Antigen Matched Versus Usual Matched approach- Experience from Tertiary Care Hospital! (2026/05/01) ♡
- Assessment of Chronic Liver Disease by Transient Elastography as a Surrogate Marker in Beta Thalassemia Major Patients. (2026/05/01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Effects of vitamin D deficiency and supplementation on myocardial and hepatic iron status and function in children with beta-thalassemia. (2026/04/14) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Mechanisms of resistance to Tmprss6 antisense oligonucleotides in a mouse model of β-thalassemia. (2026/04/09) ♡
- Meta-analysisiAll studies on one question combined and calculated together. This is the strongest form of research that exists: a single loose study can be coincidence, dozens together much less so. The label says something about the design, not about the outcome — which can also be that something does NOT work. Burden of transfusion-transmitted infections among patients with thalassaemia and sickle cell anaemia in India: A systematic review and meta-analysis. (2026/04/01) ♡
- The Application of Machine-Learning Algorithms for Multiclass Classification of Microcytic Anemia Revealed That a Minimum Required Number of Hematological Parameters Is Enough to Achieve High Diagnostic Accuracy. (2026/04/01) ♡
- Impact of SNP Variants in PON-1 or UGT1A1 on Iron Chelation Therapy Outcomes and Zinc Status in Thalassemia Major Patients. (2026/04/01) ♡
- Haemoglobinopathies and health disparities: findings of a large-scale sample survey among indigenous populations in Odisha, India. (2026/03/27) ♡
- 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. Synergistic Potential of Thalidomide and Hydroxyurea in Sickle Cell Disease Management: A Promising Combination Therapy. (2026/03/01) ♡
- 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. Molecular Genetics and Recent Management Strategies of HB E-β Thalassemia. (2026/03/01) ♡
- 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. Emerging Considerations in Transfusion Medicine: Hematopoietic Stem and Progenitor Cell Collection for Gene Therapy for Sickle Cell Disease and Transfusion-Dependent Thalassemia. (2026/03/01) ♡
- Vitamin B12 Deficiency Does Not Induce Homocysteine Increase in Transfusion-Dependent β-Thalassemia Major Patients Receiving Folic Acid Supplementation. (2026/03/01) ♡
- Diagnostic Accuracy of the Patra Index and Red Cell Distribution Width Index in Distinguishing Beta-Thalassemia Trait From Iron Deficiency Anemia: A Retrospective Comparative Study. (2026/02/28) ♡
- 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. The Burden of Thalassemia and Various Hemoglobinopathies in Bihar: Trends and Patterns. (2026/02/04) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Exploring a Case of Unconjugated Hyperbilirubinaemia Resulting from the Rare Coexistence of Inherited Disorders- A Case Report. (2026/02/01) ♡
- Discovery of a Novel DNMT1 Inhibitor with Improved Efficacy in Treating β-Thalassemia. (2026/02/01) ♡
- Identification of RBM3 as a novel regulator of human fetal hemoglobin expression. (2026/02/01) ♡
- Interpretable machine learning models for beta thalassemia prediction: an explainable AI approach for smart healthcare 5.0. (2026/01/14) ♡
- Thalassemia does not significantly affect embryo ploidy outcomes in women undergoing IVF with preimplantation genetic testing. (2026/01/13) ♡
- 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. Current research status of third-generation sequencing technology in thalassemia detection. (2026/01/13) ♡
- Biventricular and bia-trial strain by cardiac magnetic resonance in thalassaemia intermedia: patterns and correlates. (2026/01/01) ♡
- Reproductive Considerations of Gene Therapies for Sickle Cell Disease and Beta-Thalassemia. (2026/01/01) ♡
- 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. Targeting LncRNAs with CRISPR/Cas9 for Kidney Therapeutics: A Review. (2026/01/01) ♡
- Role of Serum Apelin and Carotid Artery Intima Thickness in Predicting Early Atherosclerotic Changes in Children With Beta-Thalassemia Major. (2026/01/01) ♡
- Effect of Closed Kinetic Chain Exercise Via Telerehabilitation on Muscle Strength, Balance, Fatigue and Hemoglobin Levels in Beta Thalassemia Major Children: A Randomized Controlled Trial. (2026/01/01) ♡
- HbE/β-thalassemia presenting with Moyamoya angiopathy-associated ischemic stroke and noncompressive intrathoracic extramedullary hematopoiesis. (2026/01/01) ♡
- Single-Position Peptide Clustering for Peptidomics Reveals Novel Disease Biomarkers and Dysregulated Proteolytic Characteristics. (2026/01/01) ♡
- 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. A Long-term Follow-up Study in Participants Who Received CTX001 (2026-08-11) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants (2026-08-07) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety and Efficacy of Hemoglobin F Inducers in Patients With Beta Thalassemia (2026-08-06) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease (2026-08-04) ♡
- 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. A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia (2026-08-03) ♡
- 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. A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) (2026-08-03) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell Disease (2026-07-31) ♡
- 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. A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT) (2026-07-31) ♡
- 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. A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) (2026-07-31) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. The Longitudinal Relationship of HU Adherence to HRQOL, Barriers to Adherence and Habit in SCD. (2026-07-31) ♡
- Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors (2026-07-29) ♡
- Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. Gene-Modified Stem Cell Therapy for Subjects With Transfusion-dependent Beta-thalassemia (2026-07-27) ♡
- Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease (2026-07-27) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia (2026-07-24) ♡
- Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. Gene Therapy for Patients With Transfusion-Dependent β-Thalassemia (2026-07-23) ♡
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