Scientific News
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Pain Impact and Management in Individuals With Transfusion-Dependent Thalassaemia
Over the last several decades, there has been significant progress in addressing the main symptoms and related health issues of people with transfusion-dependent thalassemia (TDT). Yet, the problem of pain…
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Chinese Base Editing Therapy ‘Clinically Cured’ Patient with β-Thalassaemia, Researchers Say
Shanghai-based biotech company CorrectSequence Therapeutics, in collaboration with Guangxi Medical University, has achieved a significant clinical milestone by successfully treating a young patient with transfusion-dependent β-thalassaemia using their base editing…
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CASGEVY | 1st Gene-Editing Therapy for Thalassaemia and SCD Approved in UK
Casgevy is the first medicine to be licensed that uses the innovative gene-editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020. The therapy, developed by…
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Imatinib’s Impact on Reducing Painful Episodes in Sickle Cell Disease
Currently, hydroxyurea (HU) is the only FDA-approved drug highly recommended for treating sickle cell anemia. However, its efficiency varies among patients with sickle cell disease for unknown reasons. Given the…
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Corticosteroid Use in Pregnant Women with SCD Doubles Risk of Severe VOCs
According to a French study, corticosteroids used during pregnancy are a risk factor for more severe vaso-occlusive crises (VOCs) in women with sickle cell disease (SCD). This risk was especially…
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The Hidden Dangers of Sickle Cell Trait: Unraveling the Risk for Complications
Worldwide, it is estimated that there are 300 million people with sickle cell trait and one-third of this number are in sub-Saharan Africa. With these staggering figures, sickle cell disease…
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Transfusion-Dependent Thalassaemia Patients Display Mild COVID-19, Weakened Antibody Response
In a recent research published in the journal Cureus, patients with transfusion-dependent thalassaemia (TDT) were shown to have moderate or asymptomatic COVID-19 manifestations. Patients with TDT had an immunoglobulin G…
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Scientists Develop Safer Method for Gene-Editing Therapy Delivery
Researchers have found a new method of delivering gene-editing medicines to persons with blood illnesses such as thalassaemia and sickle cell disease (SCD), which if used in the clinic might…
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Deferiprone Reduces Iron Overload in Young Patients With Transfusion-Dependent Beta-Thalassaemia
In young patients with transfusion-dependent beta-thalassaemia (TDT), the use of early-start deferiprone appeared to be well tolerated and was associated with efficacy in reducing iron overload, according to the results…
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Epidemiology of Clinically Significant Forms of Alpha- and Beta Thalassemia: A Global Map of Evidence and Gaps
This systematic study looked at the worldwide and birth prevalence of clinically relevant types of alpha- and beta-thalassaemia. Searches were conducted in Embase, MEDLINE, and the Cochrane Library for observational…
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