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"A mutation in the TMPRSS6 gene, encoding a transmembrane serine protease that suppresses hepcidin production, in familial iron deficiency anemia refractory to oral iron"

While this trial lacked statistical power to detect differences in cognitive and functional assessments between treated groups, GRF6019 exhibited excellent safety, feasibility, and tolerability

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Spicer J, Shewale J, Sepesi B, Antonoff M , Correa A, Hofstetter W, Rice D, Vaporciyan A, Mehran R, Walsh G, Roth J, Swisher S

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Maan JS, Rosani A, Saadabadi A