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AbstractThalassemia and sickle cell disease are inherited hemoglobinopathies caused by pathogenic variants in the globin genes and represent a major global health…

Introduction: Sickle Cell Disease (SCD) is a common inherited haemoglobinopathy with significant oral and systemic implications. Despite its prevalence in multi-…

Hematopoietic stem cell transplantation (HSCT) is both possible and effective in adult patients with sickle cell disease (SCD), according to a…

One of the $3 million 2026 Breakthrough Prizes — in Life Sciences — has been awarded to two researchers whose discoveries reshaped the treatment of sickle cell…

Nigeria now bears the world’s highest burden of sickle cell disease among children, with more than 1.5 million cases recorded in those under 15, according to a new…

Shares of Agios Pharmaceuticals AGIO declined sharply by 23% on Monday after Novo Nordisk NVO announced positive top-line data for its investigational drug,…

Novo Nordisk is planning to move ahead with regulatory filings for etavopivat, potentially a first-in-class therapy for sickle cell disease, based on positive…

A global, late-stage clinical trial testing etavopivat in people with sickle cell disease (SCD) has hit its main goals, showing that the experimental oral therapy…

Novo Nordisk is gearing up to submit its rare blood disorder drug to the FDA after the former Forma Therapeutics asset reduced a painful complication of sickle cell…

Sickle cell disease (SCD) is a monogenic disorder where autologous gene therapy may offer a safer curative alternative to allogeneic transplantation. We report…