The Administrative Core (Core A) will be managed by John Van den Anker, Program Director, and Edward Connor, Co-Program Director. Dr. Van den Anker is the Vice Chair of Pediatrics for Experimental Therapeutics at Children's National Medical Center (CNMC) and brings expertise in the management of programs such as the Pediatric Pharmacology Research Unit, and has extensive clinical and research expertise in several areas of developmental and pediatric pharmacology. He also serves as PI of Project 1 (Determine the time course of urine biomarkers for morpholino anti-sense oligonucleotides accumulation in renal epithelial cells). Dr. Connor is the Director of the Office of Innovation Development and Investigational Therapeutics and brings extensive expertise in pediatric drug development, clinical trials, clinical/translational research, and executive research management/administration and also serves as co-l of Project 1. The Co-Directors have complementary capabilities that will allow them to effectively direct the Administrative Core. This Core has two specific aims.
In Specific Aim 1, fiscal management of all Projects and Cores, oversight of balancing of Core resources, convening of the Internal Steering Committee as well as annual meetings of the External Advisory Board, and solicitation and processing of Pilot Project applications will be the responsibility of Dr. Van den Anker and his administrative team.
In Specific Aim 2, monthly conference calls of key personnel on all Projects and Cores, monitoring of research progress on each Project and Core, including timely achievement of milestones, oversight of annual scientific reports, and oversight and monitoring of human subjects and regulatory compliance will be the responsibility of Dr. Connor. Finally, both Drs. Van den Anker and Connor are tightly integrated into the CTSA efforts and this very active involvement of the CTSA within the RPDP investigations will ensure synergism with this nationally-based clinical and translational research effort.

National Institute of Health (NIH)
Eunice Kennedy Shriver National Institute of Child Health & Human Development (NICHD)
Specialized Center--Cooperative Agreements (U54)
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Special Emphasis Panel (ZHD1-DSR-A)
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Children's Research Institute
United States
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Yu, Qing; Morales, Melissa; Li, Ning et al. (2018) Skeletal, cardiac, and respiratory muscle function and histopathology in the P448Lneo- mouse model of FKRP-deficient muscular dystrophy. Skelet Muscle 8:13
Defour, Aurelia; Medikayala, Sushma; Van der Meulen, Jack H et al. (2017) Annexin A2 links poor myofiber repair with inflammation and adipogenic replacement of the injured muscle. Hum Mol Genet 26:1979-1991
Novak, James S; Hogarth, Marshall W; Boehler, Jessica F et al. (2017) Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle. Nat Commun 8:941
Smits, Anne; van den Anker, John N; Allegaert, Karel (2017) Clinical pharmacology of analgosedatives in neonates: ways to improve their safe and effective use. J Pharm Pharmacol 69:350-360
Vila, Maria C; Rayavarapu, Sree; Hogarth, Marshall W et al. (2017) Mitochondria mediate cell membrane repair and contribute to Duchenne muscular dystrophy. Cell Death Differ 24:330-342
Samiee-Zafarghandy, S; van den Anker, J N; Laughon, M M et al. (2016) Sildenafil and retinopathy of prematurity risk in very low birth weight infants. J Perinatol 36:137-40
Samardzic, Janko; Smits, Anne; Spriet, Isabel et al. (2016) Different Vancomycin Immunoassays Contribute to the Variability in Vancomycin Trough Measurements in Neonates. Biomed Res Int 2016:1974972
Hathout, Yetrib; Seol, Haeri; Han, Meng Hsuan J et al. (2016) Clinical utility of serum biomarkers in Duchenne muscular dystrophy. Clin Proteomics 13:9
Gupta, Charu; Massaro, An N; Ray, Patricio E (2016) A new approach to define acute kidney injury in term newborns with hypoxic ischemic encephalopathy. Pediatr Nephrol 31:1167-78
Hathout, Yetrib; Conklin, Laurie S; Seol, Haeri et al. (2016) Serum pharmacodynamic biomarkers for chronic corticosteroid treatment of children. Sci Rep 6:31727

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