Eteplirsen for the treatment of Duchenne muscular dystrophy von Mendell, Jerry R., Rodino-Klapac, Louise R., Sahenk, Zarife, Roush, Kandice, Bird, Loren, Lowes, Linda P., Alfano, Lindsay, Gomez, Ann Maria, Lewis, Sarah, Kota, Janaiah, Malik, Vinod, Shontz, Kim, Walker, Christopher M., Flanigan, Kevin M., Corridore, Marco, Kean, John R., Allen, Hugh D., Shilling, Chris, Melia, Kathleen R., Sazani, Peter, Saoud, Jay B., Kaye, Edward M.

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A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy von Mendell, Jerry R, Sahenk, Zarife, Malik, Vinod, Gomez, Ana M, Flanigan, Kevin M, Lowes, Linda P, Alfano, Lindsay N, Berry, Katherine, Meadows, Eric, Lewis, Sarah, Braun, Lyndsey, Shontz, Kim, Rouhana, Maria, Clark, Kelly Reed, Rosales, Xiomara Q, Al-Zaidy, Samiah, Govoni, Alessandra, Rodino-Klapac, Louise R, Hogan, Mark J, Kaspar, Brian K

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Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes von Mendell, Jerry R., Sahenk, Zarife, Al-Zaidy, Samiah, Rodino-Klapac, Louise R., Lowes, Linda P., Alfano, Lindsay N., Berry, Katherine, Miller, Natalie, Yalvac, Mehmet, Dvorchik, Igor, Moore-Clingenpeel, Melissa, Flanigan, Kevin M., Church, Kathleen, Shontz, Kim, Curry, Choumpree, Lewis, Sarah, McColly, Markus, Hogan, Mark J., Kaspar, Brian K.

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AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models von Sondergaard, Patricia C., Griffin, Danielle A., Pozsgai, Eric R., Johnson, Ryan W., Grose, William E., Heller, Kristin N., Shontz, Kim M., Montgomery, Chrystal L., Liu, Joseph, Clark, Kelly Reed, Sahenk, Zarife, Mendell, Jerry R., Rodino‐Klapac, Louise R.

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Safety and Efficacy of AAV Retrograde Pancreatic Ductal Gene Delivery in Normal and Pancreatic Cancer Mice von Quirin, Kayla A, Kwon, Jason J, Alioufi, Arafat, Factora, Tricia, Temm, Constance J, Jacobsen, Max, Sandusky, George E, Shontz, Kim, Chicoine, Louis G, Clark, K Reed, Mendell, Joshua T, Korc, Murray, Kota, Janaiah

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Human α7 Integrin Gene (ITGΑ7) Delivered By Adeno-Associated Virus Reverses The Phenotype Of The Double Knock Out (DKO) Mouse Devoid Of Dystrophin And Utrophin (P5.099) von Heller, Kristin, Montgomery, Chrystal, Shontz, Kim, Janssen, Paul, Clark, Reed, Mendell, Jerry, Rodino-Klapac, Louise

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609. Systemic Delivery of Dysferlin Overlap Vectors Mediates Functional Recovery of Dysferlin Deficiency von Sondergaard, Patricia C., Griffin, Danielle A., Pozsgai, Eric R., Johnson, Ryan, Shontz, Kim, Mendell, Jerry R., Rodmo-Klapac, Louise R.

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Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice von Martin, Paul T, Xu, Rui, Rodino-Klapac, Louise R, Oglesbay, Elaine, Camboni, Marybeth, Montgomery, Chrystal L, Shontz, Kim, Chicoine, Louis G, Clark, K. Reed, Sahenk, Zarife, Mendell, Jerry R, Janssen, Paul M. L

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Eteplirsen, a Phosphorodiamidate Morpholino Oligomer (PMO) for Duchenne Muscular Dystrophy (DMD): Clinical Update and Longitudinal Comparison to External Controls on Six-Minute Wal... von Mendell, J., Goemans, Nathalie, Rodino-Klapac, Louise, Sahenk, Zarife, Lowes, Linda, Alfano, Lindsay, Berry, K, Peterson, E, Lewis, S, Shontz, Kim, Shao, J, Duda, P, Donoghue, C, Schnell, F, Dworzak, J, Wentworth, Bruce, Kaye, E., Mercuri, E, Italian Network, DMD

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Dual Vector Co-Delivery of nNOS-Recruiting Minidystrophin (ΔH2-R15/ΔR18-19) as a Therapeutic Strategy for DMD (S05.007) von Malik, Vinod, Shontz, Kim, Montgomery, Chrystal, Rodino-Klapac, Louise, Mendell, Jerry

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C-2. Eteplirsen, a Phosphorodiamidate Morpholino Oligomer (PMO) for the Treatment of Duchenne Muscular Dystrophy (DMD): 168 Week Update on Six-Minute Walk Test (6MWT), Pulmonary Fu... von Mendell, Jerry R., Rodino-Klapac, Louise, Sahenk, Zarife, Rouch, Kandice, Bird, Lauren, Lowes, Linda, Alfano, Lindsay, Berry, Katherine, Lewis, Sarah, Shontz, Kim, Flanigan, Kevin, Shilling, Christopher, Duda, Petra, Saoud, Jay

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Baseline Performance on 6MWT Correlates with Other Functional Outcomes in a Phase IIb Study of Exon-Skipping Antisense Oligonucleotide Eteplirsen for Use in Duchenne Muscular Dystr... von Saoud, Jihad, Mendell, Jerry, Rodino-Klapac, Louise, Sahenk, Zarife, Roush, Kandice, Bird, Loren, Lowes, Linda, Alfano, Lindsay, Gomez Ramirez, Ana Maria, Lewis, Sarah, Malik, Vinod, Shontz, Kim, Shilling, Christopher, Sazani, Peter, Kaye, Edward

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Safety Profile and Pharmacokinetic Properties of Eteplirsen in the Treatment of Boys with Duchenne Muscular Dystrophy (S6.001) von Saoud, Jihad, Mendell, Jerry, Rodino-Klapac, Louise, Sahenk, Zarife, Roush, Kandice, Bird, Loren, Lowes, Linda, Alfano, Lindsay, Gomez Ramirez, Ana Maria, Lewis, Sarah, Malik, Vinod, Shontz, Kim, Shilling, Christopher, Sazani, Peter, Kaye, Edward

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Results at 96 Weeks of a Phase IIb Extension Study of the Exon-Skipping Drug Eteplirsen in Patients with Duchenne Muscular Dystrophy (DMD) (S6.002) von Kaye, Edward, Mendell, Jerry, Rodino-Klapac, Louise, Sahenk, Zarife, Roush, Kandice, Bird, Loren, Lowes, Linda, Alfano, Lindsay, Gomez Ramirez, Ana Maria, Lewis, Sarah, Malik, Vinod, Shontz, Kim, Shilling, Christopher, Sazani, Peter, Saoud, Jihad

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A Potential microRNA-Based Therapy for Pancreatic Ductal Adenocarcinoma Using AAV-Mediated Delivery von Kota, Janaiah, Chivukula, Raghu R, Kent, Oliver A, Shontz, Kim M, Maitra, Anirban, Clark, K Reed, Mendell, Jerry R, Mendell, Joshua T

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Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice von MARTIN, Paul T, RUI XU, MENDELL, Jerry R, JANSSEN, Paul M. L, RODINO-KLAPAC, Louise R, OGLESBAY, Elaine, CAMBONI, Marybeth, MONTGOMERY, Chrystal L, SHONTZ, Kim, CHICOINE, Louis G, CLARK, K. Reed, SAHENK, Zarife

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A multiobjective mesh optimization framework for mesh quality improvement and mesh untangling von Kim, Jibum, Panitanarak, Thap, Shontz, Suzanne M.

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A hybrid mesh deformation algorithm using anisotropic PDEs and multiobjective mesh optimization von Kim, Jibum, Miller, Brian J., Shontz, Suzanne M.

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A multiobjective mesh optimization framework for mesh quality improvement and mesh untangling von Kim, Jibum, Panitanarak, Thap, Shontz, Suzanne M.

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A hybrid mesh deformation algorithm using anisotropic PDEs and multiobjective mesh optimization von Kim, Jibum, Miller, Brian J., Shontz, Suzanne M.

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