Michael Pacini
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View article: Translating an HPFH-Mechanism into Oral Small Molecule Therapy for Beta-Hemoglobinopathies: Clinical Proof-of-Principle
Translating an HPFH-Mechanism into Oral Small Molecule Therapy for Beta-Hemoglobinopathies: Clinical Proof-of-Principle Open
Introduction: Hereditary persistence of fetal hemoglobin (HPFH) down-modulates co-inherited sickle cell disease (SCD) severity, encouraging characterization of HPFH-mechanisms for interventional reprise. One HPFH mechanism is polymorphisms…
Oral tetrahydrouridine and decitabine for non-cytotoxic epigenetic gene regulation in sickle cell disease: A randomized phase 1 study Open
ClinicalTrials.gov, NCT01685515.
Phase I trial of p28 (NSC745104), a non-HDM2-mediated peptide inhibitor of p53 ubiquitination in pediatric patients with recurrent or progressive central nervous system tumors: A Pediatric Brain Tumor Consortium Study Open
This phase I study demonstrated that p28 is well-tolerated in children with recurrent CNS malignancies at the adult recommended phase II dose.