G.P.302 von Peay, H.L, Scharff, H, Biesecker, B.B, Wilfond, B.S, Johnson, J, Escolar, D.M, Bowie, J, Nagaraju, K, Piacentino, J, Tibben, A

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G.P.303 von Peay, H.L, Scharff, H, Biesecker, B.B, Wilfond, B.S, Johnson, J, Escolar, D.M, Nagaraju, K, Piacentino, J, Bowie, J, Tibben, A

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P.11.14 Phase 1b single and multiple ascending dose study to evaluate the safety, tolerability and pharmacokinetics of HT-100 in DMD von Escolar, D.M, Davis-Golden, J, Loewy, J, Bush, E, Dykstra, K, Blaustein, M

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P.13.13 Electrical impedance myography in DMD: A multi-center study of reliability and relationships to strength and function von Zaidman, C, Bohorquez, J, Wang, L, Florence, J, Connolly, A.M, Escolar, D.M, Williams, G

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Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy von ESCOLAR, D. M, HACHE, L. P, PESTRONK, A, RYAN, M. M, MONASTERIO, E, DAY, J. W, ZIMMERMAN, A, ARRIETA, A, HENRICSON, E, MAYHEW, J, FLORENCE, J, HU, F, CLEMENS, P. R, CONNOLLY, A. M, CNAAN, A, MCDONALD, C. M, VISWANATHAN, V, KORNBERG, A. J, BERTORINI, T. E, NEVO, Y, LOTZE, T

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96. Placebo-controlled study of alglucosidase alfa in adults with pompe disease von Clemens, P.R, van der Ploeg, A, Corzo, D, Florence, J, Laforet, P, Lake, S, Pestronk, A, Rosenbloom, B, Skrinar, A, Wasserstein, M, Leshner, R, Escolar, D.M, Mayhew, J

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T.P.1.01 Use of the 6 min walk test as an endpoint in clinical trials for neuromuscular diseases von Florence, J.M, van der Ploeg, A, Clemens, P.R, Escolar, D.M, Laforet, P, Rosenbloom, B, Wasserstein, M, Skrinar, A, Pestronk, A, Mayhew, J.E

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Allogeneic stem cell transplantation corrects biochemical derangements in MNGIE von HIRANO, M, MARTI, R, SCHWARTZ, J, HAWKS, R. G, MARTONE, D. L, CAIRO, M. S, DIMAURO, S, STANZANI, M, GARVIN, J. H, SAVAGE, D. G, CASALI, C, TADESSE, S, ULDRICK, T, FINE, B, ESCOLAR, D. M, VALENTINE, M. L, NISHINO, I, HESDORFFER, C

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T.P.5.02 Pentoxifylline treatment fails to rescue muscle strength and function deterioration in prednisone-treated Duchenne muscular dystrophy (DMD) von Escolar, D.M, Gorni, K, Tesi-Rocha, A.C, Mah, J, Nevo, Y, Korengberg, A, Kolski, H, Bertorini, T, Connolly, A, Kuntz, N, Zimmerman, A, Morgenroth, L, Arrieta, A, Mayhew, J, Florence, J, Nei, L, Hu, F, Henricson, E, Leshner, R, Dubrovsky, A

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Pediatric sciatic neuropathies: A 30-year prospective study von SRINIVASAN, J, RYAN, M. M, ESCOLAR, D. M, DARRAS, B, JONES, H. R

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Pentoxifylline as a rescue treatment for DMD: A randomized double-blind clinical trial von ESCOLAR, D. M, ZIMMERMAN, A, NEVO, Y, TESI-ROCHA, C, NAGARAJU, K, RAYAVARAPU, S, HACHE, L. P, MAYHEW, J. E, FLORENCE, J, HU, F, ARRIETA, A, HENRICSON, E, BERTORINI, T, LESHNER, R. T, MAH, J. K, CLEMENS, P. R, CONNOLLY, A. M, MESA, L, GORNI, K, KORNBERG, A, KOLSKI, H, KUNTZ, N

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G.P.302: Perceived barriers and facilitators of participation in clinical trials for Duchenne and Becker muscular dystrophy von Peay, H.L., Scharff, H., Biesecker, B.B., Wilfond, B.S., Johnson, J., Escolar, D.M., Bowie, J., Nagaraju, K., Piacentino, J., Tibben, A.

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G.P.303: Influences on parental decision making about participation in Duchenne and Becker muscular dystrophy clinical trials von Peay, H.L., Scharff, H., Biesecker, B.B., Wilfond, B.S., Johnson, J., Escolar, D.M., Nagaraju, K., Piacentino, J., Bowie, J., Tibben, A.

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Liquid formulation of pentoxifylline is a poorly tolerated treatment for duchenne dystrophy von Zimmerman, Angela, Clemens, Paula R., Tesi-Rocha, Carolina, Connolly, Anne, Iannaccone, Susan T., Kuntz, Nancy, Arrieta, Adrienne, Hache, Lauren, Henricson, Erik, Hu, Fengming, Mayhew, Jill, Escolar, Diana M.

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T.P.1.01 Use of the 6min walk test as an endpoint in clinical trials for neuromuscular diseases von Florence, J.M., van der Ploeg, A., Clemens, P.R., Escolar, D.M., Laforet, P., Rosenbloom, B., Wasserstein, M., Skrinar, A., Pestronk, A., Mayhew, J.E.

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G.P.1 01 Clinical presentation and disease progression in late-onset Pompe disease von Escolar, D.M., Wokke, J.H.J., Pestronk, A., Carter, G.T., Jaffe, K.M., Laforêt, P.

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G.P.10 07 CINRG pilot trial of oxatomide in steroid-naive Duchenne muscular dystrophy von Buyse, G.M., Escolar, D.M., Goemans, N., Henricson, E., van den Hauwe, M., Jara Vallejos, A., Shao, C., Patel, K.M., McCarter, R., Leshner, R., Florence, J., Mayhew, J.

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Barriers and facilitators to clinical trial participation among parents of children with pediatric neuromuscular disorders von Peay, H.L., Biesecker, B.B., Wilfond, B.S., Jarecki, J., Umstead, K.L., Escolar, D.M., Tibben, A.

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PIP(2) binding residues of Kir2.1 are common targets of mutations causing Andersen syndrome von Donaldson, M.R., Jensen, J.L., Tristani-Firouzi, M., Tawil, R., Bendahhou, S., Suarez, W.A., Cobo, A.M., Poza, J.J., Behr, E., Wagstaff, J., Szepetowski, P., Pereira, S., Mozaffar, T., Escolar, D.M., Fu, Y.H., Ptacek, L.J.

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