Search Results - "Powers, Brenna"

Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes by 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

“…Historically, autosomal recessive 5q-linked spinal muscular atrophy (SMA) has been the leading inherited cause of infant death. SMA is caused by the absence of…”
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Natural History of Steroid-Treated Young Boys With Duchenne Muscular Dystrophy Using the NSAA, 100m, and Timed Functional Tests by 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.

“…Clinical trials targeting younger cohorts of boys with Duchenne muscular dystrophy are necessary as earlier intervention may maximize treatment effect. Boys…”
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Validity and Reliability of the Neuromuscular Gross Motor Outcome by 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.

“…Approved treatments in spinal muscular atrophy (SMA) have resulted in unprecedented gains for many individuals. Use of available outcomes, typically developed…”
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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 by 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) by 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.

“…Abstract only…”
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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) by 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.

“…Abstract only…”
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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 by 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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eP212: Safety, β-Sarcoglycan expression, and functional outcomes from systemic gene transfer of rAAVrh74.MHCK7.hSGCB in LGMD2E/R4 by 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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LATE BREAKING NEWS ORAL PRESENTATION by 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: 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 by 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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