6. Characterization of Cardiac Function in Subjects with Hereditary Hemochromatosis Who Are New York Heart Association Functional Class I (Douglas R. Rosing, M.D.) Due to the relative unavailability of the primary co-investigator Dr. Yukitaka Shizukuda, relatively little data analysis occurred during the past year. However, increased availability of Dr. Shizukuda should result in further analysis of the available data as well as a decision made as to whether it is worth undertaking a 10 year reactivation of the protocol. We have submitted an amendment to re-evaluate this population at the 10 year or present follow up date for the occurrence of atrial fibrillation and other clinically significant arrhythmias. 7. Retrospective Evaluation of Liver Disease in Patients with Heart Disease (Douglas R. Rosing, M.D.) This project is being undertaken in collaboration with Liver Disease Branch of NIDDK and involves a retrospective analysis through chart review of cardiac disease on liver function. Sixty-seven subjects have been analyzed, but little progress has been made in the last year due to difficulty in gaining access to medical records of candidates. Hopefully this problem will be rectified in the coming year, and the project will be completed.
| Shamburek, Robert D; Bakker-Arkema, Rebecca; Auerbach, Bruce J et al. (2016) Familial lecithin:cholesterol acyltransferase deficiency: First-in-human treatment with enzyme replacement. J Clin Lipidol 10:356-67 |
| Shamburek, Robert D; Bakker-Arkema, Rebecca; Shamburek, Alexandra M et al. (2016) Safety and Tolerability of ACP-501, a Recombinant Human Lecithin:Cholesterol Acyltransferase, in a Phase 1 Single-Dose Escalation Study. Circ Res 118:73-82 |
