Catherine Skari
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Sep- 2026 -8 SeptemberScientific Literature
Patient-Reported Outcomes With Luspatercept Through 5 Years of Treatment in Patients With Non-Transfusion-Dependent β-Thalassemia Treated in the BEYOND Trial
Abstract In the phase 2, double‐blind, randomized controlled BEYOND trial (NCT03342404), luspatercept increased hemoglobin levels in patients with non‐transfusion‐dependent β‐thalassemia…
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8 SeptemberScientific Literature
Modelling the Cost-Effectiveness of Non-Invasive Prenatal Testing in the English Sickle Cell and Thalassaemia Screening Pathway
Background: Sickle cell disease (SCD) and thalassaemia are inherited conditions causing chronic anaemia, increased infection risk, and multi-organ failure. Standard…
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8 SeptemberNews
Prevalence of Severe Thalassemia and Performance of Prenatal Screening Tests Among Pregnant Women at Siriraj Thalassemia Center in Thailand
Abstract Severe thalassemia remains a significant public health concern in Southeast Asia. Prenatal screening is an effective strategy for early…
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8 SeptemberScientific Literature
Systematic Review of Economic Evaluations in Thalassaemia Screening Programmes Globally: Developing Guidance for Low- and Middle-Income (LMIC) Settings
Objectives Thalassaemia, a genetic blood disorder, is a major public health burden. Most affected individuals reside in low-and-middle-income countries (LMICs).…
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8 SeptemberScientific Literature
Outcomes With Luspatercept in Patients With Β-Thalassemia: A Systematic Review and Metaanalysis
Abstract Background: β-thalassaemia is a genetic blood disorder marked by ineffective erythropoiesis and chronic anaemia, often requiring lifelong red blood cell…
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7 SeptemberScientific Literature
Evaluating Mitapivat for the Treatment of Alpha or Beta Thalassemia
INTRODUCTION Thalassemia is a group of diverse genetic disorders with worldwide distribution that affect hemoglobin synthesis. Until recently, the therapeutic…
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7 SeptemberScientific Literature
Phase 2a Randomized Study to Evaluate Sapablursen in Patients With Non-Transfusion Dependent Β-Thalassemia Intermedia
Non-transfusion-dependent β-thalassemia intermedia (NTDT) is a genetically heterogeneous disorder characterized by decreased β-globin production in erythroid cells resulting from mutations…
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7 SeptemberScientific Literature
2026 Update on Clinical Trials in β-Thalassemia
Abstract The therapeutic landscape of β-thalassemia has evolved rapidly over the past decade, shifting from a historical reliance on transfusion…
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7 SeptemberScientific Literature
Long-Term Efficacy and Safety Results of Betibeglogene Autotemcel Gene Therapy for Transfusion-Dependent Β-Thalassemia
Abstract Betibeglogene autotemcel (beti-cel) gene therapy for transfusion-dependent β-thalassemia (TDT) involves autologous transplantation of hematopoietic stem and progenitor cells transduced…
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7 SeptemberScientific Literature
Exa-cel in Children with Transfusion-Dependent β-Thalassemia or Sickle Cell Disease
Background Exagamglogene autotemcel (exa-cel) is a cell therapy in which autologous CD34+ hematopoietic cells are engineered through ex vivo clustered…
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