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To assess whether non-myeloablative hematopoietic cell transplantation can affect the progression of multi-organ damage in a preclinical sickle cell disease model.
To evaluate whether frequent home-based hemoglobin monitoring using nailbed optical measurement via a smartphone application could identify physiologic trends associated with the onset of vaso-occlusive crises in adults with sickle...
To evaluate whether palmitoylethanolamide (PEA), a non-intoxicating lipid mediator related to cannabinoid signaling, could reduce pain hypersensitivity and inflammatory activity in sickle cell disease (SCD).
To evaluate whether a structured Project ECHO virtual learning series could improve healthcare professionals’ knowledge and confidence in facilitating transition from pediatric to adult care for patients with sickle cell...
To evaluate the efficacy and safety of etavopivat, a selective activator of red blood cell pyruvate kinase, in patients with sickle cell disease (SCD). Etavopivat improves red cell energy metabolism...
To evaluate whether a structured Project ECHO virtual learning series could improve healthcare professionals’ knowledge and confidence in facilitating transition from pediatric to adult care for patients with sickle cell...
To evaluate soluble C5b-9 (sC5b-9), a marker of terminal complement activation, as a biomarker for hemolysis-associated complications in sickle cell disease (SCD).
To investigate how placental pathology contributes to adverse pregnancy outcomes in sickle cell disease (SCD).
To evaluate whether non-genotoxic antibody-drug conjugate (ADC) conditioning can support donor hematopoietic engraftment in a murine sickle cell disease (SCD) transplant model.
To assess safety, pharmacokinetics (PK), pharmacodynamics (PD), and biologic activity of pociredir in patients with SCD, including effects on fetal hemoglobin (HbF) and hemolysis.