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Focus on rare disease "thalassemia": FDA awards Mitapivat orphan drug title

 Last Update: 2020-06-24
 Source: Internet
 Author: User

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Pharmaceutical company Agios announced today that the U.SFood and Drug Administration, theAdministration of(
FDA) has awarded acetone acid kinase-R (PKR) activator mitapivat for the treatment of thalassemia orphan drug titleMitapivat is a research small molecular deconstruction activator of wild and multi-mutant PKRDr Chris Bowden, Chief Medical Officer of Agios, said: "The acquisition of the orphan drug designation is an important milestone for AgiosWe will continue to promote Mitapivat therapy for thalassemia patientsThe latest data from the Mitapivat treatment of alpha and beta thalassemia II will be presented at the upcoming annual meeting of the European Society of Hematology (EHA)thalassemias is an inherited blood disorder that causes hemoglobin synthesis disorders, and its symptoms can vary according to type, to varying degrees from asymptomatic to severeCommonly thalassemia is accompanied by a typical symptom of anemia, namely low levels of red blood cellsAnemia can lead to feelings of fatigue and pale skin tone, as well as bone disease, spleen swelling, jaundice, dark urine, andgrowth delays in children

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