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Gdf -11 a new target to improve anemia in thalassemia.
β-thalassemias are characterized by ineffective red blood cell (RBC) production, leading to anemia, iron overload, and organ failure. As current treatment options for β-thalassemia are limited, there is a clear unmet need for alternative therapies.
Hematology and oncology, pacemakers for EU HTA
EHA’s Martin Kaiser, right, addressing his fellow panelists. Left to right: Brian Cuffel (Bayer), Caroline Pothet (EMA), Roisin Adams (HTA CG), Bernhard Wörmann (DGHO) and Elisabeth de Vries (ESMO).
Read moreAddressing immunoglobulin shortages: EHA and EBA’s strategic recommendations at the EMA
In recent years, shortages of immunoglobulins (Ig) have become a growing concern in the European Union (EU). Since 2018, the European Medicines Agency (EMA)—the EU agency that evaluates and supervises medicines—has received an increasing number of shortage reports.
Read moreNew GFI1B variants in bleeding and platelet disorders
Platelets restrict blood loss upon vessel damage by formation of a clot (thrombus). Recently, we reported a family with a bleeding and platelet disorder (BPD), which was caused by a defect in the gene GFI1B1.
Read moreSelected EMA news
February 2025New medicines recommended for approval
Dyrupeg (pegfilgrastim) – biosimilar
Treatment to shorten the duration of neutropenia and help prevent febrile neutropenia after cytotoxic chemotherapy
Eltrombopag Accord (eltrombopag) – generic
Treatment of primary immune thrombocytopenia and thrombocytopenia associated with chronic hepatitis C
New information on…
Press Release: Better funding for research into blood diseases will save more lives
“Haematology is probably the area of medicine that has progressed the most in recent years”, said Professor Robin Foà of “La Sapienza” University of Rome.
Read moreAdvances from genome sequencing are paving the way to personalized treatment for leukemia and pre-leukemia
In acute myeloid leukemia (AML) our understanding of how modifications to DNA molecules that do not result in sequence change (i. e.
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