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Safety and early hints of benefit seen in phase 1b trial of PF-04447943
Sickle cell disease (SCD) patients were safely treated with a phosphodiesterase 9A (PDE9) inhibitor therapy called PF-04447943 with no serious side effects reported and possible evidence of fewer vaso-occlusion pain crises, a Phase 1b clinical trial reports.
Safety and early signs of possible efficacy — including a drop in biomarkers linked to vaso-occlusion — were seen regardless of whether patients given PF-0444794 3 were also taking hydroxyurea.
The study, “PF-04447943, a phosphodiesterase 9A Inhibitor, in Stable Sickle Cell Disease Patients: A Phase Ib Randomized, Placebo-Controlled Study” was published in the journal, Clinical and Translation Science.
Sickle cell disease, caused by an inherited alteration in a gene that instructs the making of the beta-globin subunit of hemoglobin, the protein responsible for carrying oxygen in the blood, leads to sickle hemoglobin production and sickle-shaped red blood cells.
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.