Earlier this year, the U.S. Food and Drug Administration (FDA) approved mitapivat (PYRUKYND) for the treatment of patients with hemolytic anemia due to pyruvate kinase deficiency (PKD). According to 2…
Rocket Pharmaceuticals, Inc. ("Rocket") has dedicated its mission to developing gene therapy solutions for rare childhood conditions. Recently, the company shared the publication of preliminary data from a Phase…
An investigative therapy for hemolytic disease of the fetus and newborn (HDFN) has just received the FDA's Rare Pediatric Disease and Orphan Drug Designations. This therapy is called nipocalimab and…
Global Blood Therapeutics (GBT) will submit their Marketing Authorization Application (MAA) for Oxbryta to the EMA by 2021, says a recent BioSpace press release. Ultimately, the MAA will seek regulatory…
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