Archive for February 2010

Over-expression of gene regulator spurs development of leukemia in babies with Down syndrome

Between 5 and 10 percent of babies with Down syndrome develop a transient form of leukemia that usually resolves on its own. However, for reasons that haven’t been clear, 20 to 30 percent of these babies progress to a more serious leukemia known as Down syndrome acute megakaryoblastic leukemia (DS-AMKL), which affects the blood progenitor [...]



FDA and European Medicines Agency Agree to Accept a Single Orphan Drug Designation Annual Report

In recognition of World Rare Disease Day, the U.S. Food and Drug Administration and the European Medicines Agency (EMA) today announced a more streamlined process to help regulators better identify and share information throughout the development process of orphan drug and biologic products, which are developed specifically to treat rare medical conditions. Both agencies have [...]



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