Scientific News
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THE HIDDEN RISK | Worsening Anaemia Linked to Pregnancy in Individuals with β-Thalassaemia Minor
β-thalassaemia minor is a genetic blood disorder that typically causes mild anaemia which typically does not require medical treatment. However, this study suggests that pregnant women with this condition may…
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HEALTH TECHNOLOGY ASSESSMENT | EU Implements New Rules to Offer Quicker Access to Advanced Medicines
The new rules establish an EU-wide framework for assessing health technologies by promoting collaboration and coordination among EU Member States. The goal is to help national authorities make more timely…
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WHAT YOU NEED TO KNOW | EMA Recommends Suspension of Pfizer’s Sickle Cell Disease Drug Oxbryta
Oxbryta, which was designed to treat sickle cell disease, has been a crucial medication for patients with mild-to-moderate symptoms. Approved in the US under an accelerated process in 2019 and…
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Stroke Risk Rises in People with Sickle Cell Disease Despite Treatment Guidelines
A new study reveals that stroke rates are continuing to rise among both adults and children living with sickle cell disease (SCD), despite established standards of care aimed at reducing…
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How to Manage Thalassaemia During Pregnancy: Expert Advice from the BSH
A comprehensive multidisciplinary approach is essential for managing conception and pregnancy in patients with a variety of medical conditions, including obesity, diabetes, and hypertension. Thalassaemia, an inherited blood disorder that…
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High Survival Rates for SCD Patients with Groundbreaking Transplant Approach
A bone marrow transplant strategy that avoids high-dose chemotherapy to prevent an immune reaction against donor stem cells has demonstrated high rates of event-free and overall survival in sickle cell…
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Oxbryta Shows Promise in Reducing Cell Sickling in Pediatric SCD Patients
In a promising leap forward for pediatric sickle cell disease (SCD) treatment, the oral medication Oxbryta (voxelotor) has been shown to reduce the sickling and disintegration of red blood cells…
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Recent Study Links Transfusion-Dependent Thalassaemia to Adverse Maternal, Neonatal Outcomes
A meta-analysis of 15 studies found that pregnant women with β-thalassaemia major and β-thalassaemia intermedia were at greater risk of heart failure, venous thromboembolism, death, and other adverse neonatal and…
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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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