Thalassemia

Early Screening Crucial to Manage Rare Genetic Disorders, Health News, ET HealthWorld

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Early Screening Crucial to Manage Rare Genetic Disorders, Health News, ET HealthWorld

by Dr Mayank Nilay Rare genetic disorders, often referred to as orphan diseases, are conditions that affect a small percentage of the population. These disorders, while individually rare, collectively impact millions of people worldwide. Early diagnosis and intervention are crucial for managing these conditions effectively. Over the years, significant advancements in medical research and technology … Read more

New report highlights urgent need to improve thalassemia awareness among Azerbaijan women

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New report highlights urgent need to improve thalassemia awareness among Azerbaijan women

5.2% of the global population carry hemoglobin abnormalities, resulting in 300,000 to 400,000 children born with severe hemoglobinopathies annually. Thalassemia, a hereditary hemoglobinopathy, occurs in 4.4 out of every 10,000 live births, and is prevalent in Mediterranean coastal areas, Africa, the Middle East, Southeast Asia, and southern China. To facilitate greater understanding of thalassemia, a … Read more

Breakthrough treatments face manufacturing and efficacy

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Breakthrough treatments face manufacturing and efficacy

In a recent review published in Gene Therapy, a group of authors explored the progress and persistent hurdles in gene therapy for inherited blood disorders, malignancies via chimeric antigen receptors (CAR)-T cells, and varied diseases treated with in vivo adeno-associated virus (AAV) vectors, addressing the transformative cures and the economic and manufacturing complexities involved. Study: … Read more