Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes von Waldrop, Megan A., Karingada, Cassandra, Storey, Mike A., Powers, Brenna, Iammarino, Megan A., Miller, Natalie F., Alfano, Lindsay N., Noritz, Garey, Rossman, Ian, Ginsberg, Matthew, Mosher, Kathryn A., Broomall, Eileen, Goldstein, Jessica, Bass, Nancy, Lowes, Linda P., Tsao, Chang-Yong, Mendell, Jerry R., Connolly, Anne M.

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Natural History of Steroid-Treated Young Boys With Duchenne Muscular Dystrophy Using the NSAA, 100m, and Timed Functional Tests von Miller, Natalie F., Alfano, Lindsay N., Iammarino, Megan A., Connolly, Anne M., Moore-Clingenpeel, Melissa, Powers, Brenna R., Tsao, Chang-Yong, Waldrop, Megan A., Flanigan, Kevin M., Mendell, Jerry R., Lowes, Linda P.

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Validity and Reliability of the Neuromuscular Gross Motor Outcome von Alfano, Lindsay N., Iammarino, Megan A., Reash, Natalie F., Powers, Brenna R., Shannon, Kiana, Connolly, Anne M., Waldrop, Megan A., Noritz, Garey H., Shell, Richard, Tsao, Chang-Yong, Flanigan, Kevin M., Mendell, Jerry R., Lowes, Linda P.

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The 50-M timed test as a simple, efficient and objective measure of gross motor function in CLN3 disease: A pilot study von Abreu, Nicolas J., Powers, Brenna R., Sveda, Michelle M., Peifer, Danielle E., Khurho, Zarsha, O'Neal, Matthew, Alfano, Lindsay N., Scherr, Jessica, de los Reyes, Emily C.

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Safety, β-Sarcoglycan Expression, and Functional Outcomes From Systemic Gene Transfer of rAAVrh74.MHCK7.hSGCB in LGMD2E/R4 (S23.005) von Rodino-Klapac, Louise R., Pozsgai, Eric R., Lewis, Sarah, Griffin, Danielle A., Meadows, Aaron S., Lehman, Kelly J., Church, Kathleen, Reash, Natalie F., Iammarino, Megan A., Powers, Brenna, Alfano, Lindsay N., Lowes, Linda P., Koenig, Erica, Neuhaus, Sarah, Li, Xiaoxi, Mendell, Jerry R.

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A Phase 2 Clinical Trial Evaluating the Safety and Efficacy of SRP-9001 for Treating Patients with Duchenne Muscular Dystrophy (S23.002) von Mendell, Jerry R., Shieh, Perry B., Sahenk, Zarife, Lehman, Kelly J., Lowes, Linda P., Reash, Natalie F., Iammarino, Megan A., Alfano, Lindsay N., Powers, Brenna, Woods, Jeremy D., Skura, Christy L., Mao, Howard C., Staudt, Loretta A., Potter, Rachael A., Griffin, Danielle A., Lewis, Sarah, Hu, Larry, Upadhyay, Sameer, Singh, Teji, Rodino-Klapac, Louise R.

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eP212: Safety, β-Sarcoglycan expression, and functional outcomes from systemic gene transfer of rAAVrh74.MHCK7.hSGCB in LGMD2E/R4 von O’Rourke, Erin, Rodino-Klapac, Louise, Pozsgai, Eric, Lewis, Sarah, Griffin, Danielle, Meadows, Aaron, Lehman, Kelly, Church, Kathleen, Reash, Natalie, Iammarino, Megan, Powers, Brenna, Alfano, Lindsay, Lowes, Linda, Koenig, Erica, Neuhaus, Sarah, Li, Xiaoxi, Mendell, Jerry R.

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eP201: A phase 2 clinical trial evaluating the safety and efficacy of delandistrogene moxeparvovec for treating patients with Duchenne muscular dystrophy von O'Rourke, Erin, Mendell, Jerry, Shieh, Perry, Sahenk, Zarife, Lehman, Kelly, Lowes, Linda, Reash, Natalie, Iammarino, Megan, Alfano, Lindsay, Powers, Brenna, Woods, Jeremy, Skura, Christy, Mao, Howard, Staudt, Loretta, Potter, Rachael, Griffin, Danielle, Lewis, Sarah, Upadhyay, Sameer, Singh, Teji, Rodino-Klapac, Louise

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LATE BREAKING NEWS ORAL PRESENTATION: LBO 3 Expression of apparent full-length dystrophin in skeletal muscle in a first-in-human gene therapy trial using the scAAV9.U7-ACCA vector... von Waldrop, Dr. Megan, Lawlor, Dr. Michael, Vetter, Tatyana Meyers, Frair, Emma, Beatka, Margaret, Meng, Dr. Hui, Iammarino, Megan, Powers, Brenna, Harris, Johan, Kaler, Maryann, Simmons, Dr. Tabatha, Wein, Dr. Nico, Flanigan, Dr. Kevin

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LATE BREAKING NEWS ORAL PRESENTATION von Waldrop, Dr. Megan, Lawlor, Dr. Michael, Vetter, Tatyana Meyers, Frair, Emma, Beatka, Margaret, Meng, Dr. Hui, Iammarino, Megan, Powers, Brenna, Harris, Johan, Kaler, Maryann, Simmons, Dr. Tabatha, Wein, Dr. Nico, Flanigan, Dr. Kevin

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Collaborative care for depression yields similar improvement among older and younger rural adults von Renn, Brenna N., Johnson, Morgan, Powers, Diane M., Vredevoogd, Mindy, Unützer, Jürgen

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Gender minority stress and diurnal cortisol profiles among transgender and gender diverse people in the United States von DuBois, L. Zachary, Puckett, Jae A., Jolly, Dee, Powers, Sally, Walker, Tian, Hope, Debra A., Mocarski, Richard, Huit, T. Zachary, Lash, Brenna R., Holt, Natalie, Ralston, Allura, Miles, Makinna, Capannola, A., Tipton, Clove, Eick, Geeta, Juster, Robert-Paul

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Unsaturated Fatty Acid Synthesis Is Associated with Worse Survival and Is Differentially Regulated by MYCN and Tumor Suppressor microRNAs in Neuroblastoma von Sheeter, Dennis A, Garza, Secilia, Park, Hui Gyu, Benhamou, Lorraine-Rana E, Badi, Niharika R, Espinosa, Erika C, Kothapalli, Kumar S D, Brenna, J Thomas, Powers, John T

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349-OR: Pancreas Volume Is Smaller in Individuals with Stage 1 Type 1 Diabetes (T1D) and Correlates with Disease Progression von VIROSTKO, JOHN, WILLIAMS, JONATHAN M., HILMES, MELISSA A., WRIGHT, JORDAN J., HAMMEL, BRENNA D., DU, LIPING, KANG, HAKMOOK, RUSSELL, WILLIAM E., POWERS, ALVIN C., MOORE, DANIEL J.

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Longitudinal Assessment of Pancreas Volume by MRI Predicts Progression to Stage 3 Type 1 Diabetes von Virostko, John, Wright, Jordan J, Williams, Jonathan M, Hilmes, Melissa A, Triolo, Taylor M, Broncucia, Hali, Du, Liping, Kang, Hakmook, Nallaparaju, Shreya, Valencia, Leah Galvez, Reyes, Demetra, Hammel, Brenna, Russell, William E, Philipson, Louis H, Waibel, Michaela, Kay, Thomas W H, Thomas, Helen E, Greeley, Siri Atma W, Steck, Andrea K, Powers, Alvin C, Moore, Daniel J

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Preliminary Report: Initial explorations into the feeding ecology of the invasive small Indian mongoose in the Caribbean using stable isotope analyses von deHart, Pieter A. P, Powers, Karen E, Hyzy, Brenna A

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Initial explorations into the feeding ecology of the invasive small Indian mongoose in the Caribbean using stable isotope analyses von deHart, Pieter A. P., Powers, Karen E., Hyzy, Brenna A.

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Monitoring the Status of Gray Bats (Myotis grisescens) in Virginia, 2009–2014, and Potential Impacts of White-Nose Syndrome von Powers, Karen E., Reynolds, Richard J., Orndorff, Wil, Hyzy, Brenna A., Hobson, Christopher S., Ford, W. Mark

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Soluble fms-Like tyrosine kinase 1 (sFlt1), endoglin and placental growth factor (PlGF) in preeclampsia among high risk pregnancies von Powers, Robert W, Jeyabalan, Arun, Clifton, Rebecca G, Van Dorsten, Peter, Hauth, John C, Klebanoff, Mark A, Lindheimer, Marshall D, Sibai, Baha, Landon, Mark, Miodovnik, Menachem

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