Precision medicine in the age of big data: The present and future role of large-scale unbiased sequencing in drug discovery and development von Vicini, P, Fields, O, Lai, E, Litwack, ED, Martin, A-M, Morgan, TM, Pacanowski, MA, Papaluca, M, Perez, OD, Ringel, MS, Robson, M, Sakul, H, Vockley, J, Zaks, T, Dolsten, M, Søgaard, M

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Triheptanoin treatment in patients with pediatric cardiomyopathy associated with long chain-fatty acid oxidation disorders von Vockley, J., Charrow, J., Ganesh, J., Eswara, M., Diaz, G.A., McCracken, E., Conway, R., Enns, G.M., Starr, J., Wang, R., Abdenur, J.E., Sanchez-de-Toledo, J., Marsden, D.L.

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UX007 for the treatment of long chain-fatty acid oxidation disorders: Safety and efficacy in children and adults following 24 weeks of treatment von Vockley, J., Burton, B., Berry, G.T., Longo, N., Phillips, J., Sanchez-Valle, A., Tanpaiboon, P., Grunewald, S., Murphy, E., Humphrey, R., Mayhew, J., Bowden, A., Zhang, L., Cataldo, J., Marsden, D.L., Kakkis, E.

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Diagnosis and treatment of cancer using genomics von Vockley, Joseph G, Niederhuber, John E

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DNA methylation in the pathophysiology of hyperphenylalaninemia in the PAH(enu2) mouse model of phenylketonuria von Dobrowolski, S F, Lyons-Weiler, J, Spridik, K, Vockley, J, Skvorak, K, Biery, A

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A New Genetic Disorder in Mitochondrial Fatty Acid β-Oxidation: ACAD9 Deficiency von He, M., Rutledge, S.L., Kelly, D.R., Palmer, C.A., Murdoch, G., Majumder, N., Nicholls, R.D., Pei, Z., Watkins, P.A., Vockley, J.

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Cognitive and adaptive functioning after liver transplantation for maple syrup urine disease: A case series von Shellmer, D. A., DeVito Dabbs, A., Dew, M. A., Noll, R. B., Feldman, H., Strauss, K. A., Morton, D. H., Vockley, J., Mazariegos, G. V.

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UX007 for the treatment of long chain-fatty acid oxidation disorders: Safety and efficacy in children and adults following 24weeks of treatment von Vockley, J., Burton, B., Berry, G.T., Longo, N., Phillips, J., Sanchez-Valle, A., Tanpaiboon, P., Grunewald, S., Murphy, E., Humphrey, R., Mayhew, J., Bowden, A., Zhang, L., Cataldo, J., Marsden, D.L., Kakkis, E.

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Urinary phenylacetylglutamine as dosing biomarker for patients with urea cycle disorders von Mokhtarani, M., Diaz, G.A., Rhead, W., Lichter-Konecki, U., Bartley, J., Feigenbaum, A., Longo, N., Berquist, W., Berry, S.A., Gallagher, R., Bartholomew, D., Harding, C.O., Korson, M.S., McCandless, S.E., Smith, W., Vockley, J., Bart, S., Kronn, D., Zori, R., Cederbaum, S., Dorrani, N., Merritt, J.L., Sreenath-Nagamani, Sandesh, Summar, M., LeMons, C., Dickinson, K., Coakley, D.F., Moors, T.L., Lee, B., Scharschmidt, B.F.

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Alterations in c-Myc phenotypes resulting from dynamin-related protein 1 (Drp1)-mediated mitochondrial fission von Sarin, M, Wang, Y, Zhang, F, Rothermund, K, Zhang, Y, Lu, J, Sims-Lucas, S, Beer-Stolz, D, Van Houten, B E, Vockley, J, Goetzman, E S, Anthony Graves, J, Prochownik, E V

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The human arginases and arginase deficiency von Iyer, R., Jenkinson, C. P., Vockley, J. G., Kern, R. M., Grody, W. W., Cederbaum, S.

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Cloning and Characterization of the Human Type II Arginase Gene von Vockley, Joseph G., Jenkinson, Christopher P., Shukla, Hridayabiranjan, Kern, Rita M., Grody, Wayne W., Cederbaum, Stephen D.

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Resting and total energy expenditure of patients with long-chain fatty acid oxidation disorders (LC-FAODs) von Gillingham, MG, Gregor, A, Vockley, J, Harding, CO, DeLany, JP

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P.13 - Resting and total energy expenditure of patients with long-chain fatty acid oxidation disorders (LC-FAODs) von Gillingham, MG, Gregor, A, Vockley, J, Harding, CO, DeLany, JP

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Cloning of Dimethylglycine Dehydrogenase and a New Human Inborn Error of Metabolism, Dimethylglycine Dehydrogenase Deficiency von Binzak, Barbara A., Wevers, Ron A., Moolenaar, Sytske H., Lee, Yu-May, Hwu, Wuh-Liang, Poggi-Bach, Jo, Engelke, Udo F.H., Hoard, Heidi M., Vockley, Joseph G., Vockley, Jerry

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Role of common gene variations in the molecular pathogenesis of short-chain acyl-CoA dehydrogenase deficiency von CORYDON, Morten Juhl, VOCKLEY, Jerry, LEVY, Harvey, SEWELL, Adrian Clive, ROE, Charles, MATERN, Dietrich, DASOUKI, Majed, GREGERSEN, Niels, RINALDO, Piero, RHEAD, William James, KJELDSEN, Margrethe, WINTER, Vibeke, RIGGS, Charles, BABOVIC-VUKSANOVIC, Dusica, SMEITINK, Jan, DE JONG, Jan

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Molecular basis of dimethylglycine dehydrogenase deficiency associated with pathogenic variant H109R von McAndrew, R. P, Vockley, J, Kim, J.-J. P

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CO67 Development of an Investigator Global Assessment of Severity in Propionic Acidemia von Longo, N., Sikirica, V., Grunewald, S., Moshkovich, O., Dionisi-Vici, C., Vockley, J., Bellenger, M.A., de las Heras, J., Banerjee, G., Schwartz, E.J.

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Missense variant in TPI1 (Arg189Gln) causes neurologic deficits through structural changes in the triosephosphate isomerase catalytic site and reduced enzyme levels in vivo von Roland, Bartholomew P., Richards, Kristen R., Hrizo, Stacy L., Eicher, Samantha, Barile, Zackery J., Chang, Tien-Chien, Savon, Grace, Bianchi, Paola, Fermo, Elisa, Ricerca, Bianca Maria, Tortorolo, Luca, Vockley, Jerry, VanDemark, Andrew P., Palladino, Michael J.

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Identification of differentially expressed genes in hepatocellular carcinoma and metastatic liver tumors by oligonucleotide expression profiling von Tackels‐Horne, Darci, Goodman, M. David, Williams, Amanda J., Wilson, Daniel J., Eskandari, Tara, Vogt, Lisa M., Boland, Joseph F., Scherf, Uwe, Vockley, Joseph G.

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