Report of MDA muscle disease symposium on newborn screening for Duchenne muscular dystrophy
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Conference Proceeding
Essential metabolic, anti-inflammatory, and anti-tumorigenic functions of miR-122 in liver
Hsu, Shu-Hao, Wang, Bo, Kota, Janaiah, Yu, Jianhua, Costinean, Stefan, Kutay, Huban, Yu, Lianbo, Bai, Shoumei, La Perle, Krista, Chivukula, Raghu R, Mao, Hsiaoyin, Wei, Min, Clark, K Reed, Mendell, Jerry R, Caligiuri, Michael A, Jacob, Samson T, Mendell, Joshua T, Ghoshal, Kalpana
Published in The Journal of clinical investigation (01.08.2012)
Published in The Journal of clinical investigation (01.08.2012)
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Journal Article
Eteplirsen for the treatment of Duchenne muscular dystrophy
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.
Published in Annals of neurology (01.11.2013)
Published in Annals of neurology (01.11.2013)
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Journal Article
Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy
Mendell, Jerry R., Goemans, Nathalie, Lowes, Linda P., Alfano, Lindsay N., Berry, Katherine, Shao, James, Kaye, Edward M., Mercuri, Eugenio
Published in Annals of neurology (01.02.2016)
Published in Annals of neurology (01.02.2016)
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Journal Article
Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates
Meyer, Kathrin, Ferraiuolo, Laura, Schmelzer, Leah, Braun, Lyndsey, McGovern, Vicki, Likhite, Shibi, Michels, Olivia, Govoni, Alessandra, Fitzgerald, Julie, Morales, Pablo, Foust, Kevin D, Mendell, Jerry R, Burghes, Arthur H M, Kaspar, Brian K
Published in Molecular therapy (01.03.2015)
Published in Molecular therapy (01.03.2015)
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Journal Article
Evidence-based path to newborn screening for duchenne muscular dystrophy
Mendell, Jerry R., Shilling, Chris, Leslie, Nancy D., Flanigan, Kevin M., al-Dahhak, Roula, Gastier-Foster, Julie, Kneile, Kelley, Dunn, Diane M., Duval, Brett, Aoyagi, Alexander, Hamil, Cindy, Mahmoud, Maha, Roush, Kandice, Bird, Lauren, Rankin, Chelsea, Lilly, Heather, Street, Natalie, Chandrasekar, Ram, Weiss, Robert B.
Published in Annals of neurology (01.03.2012)
Published in Annals of neurology (01.03.2012)
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Journal Article
Effects of Angiotensin-Converting Enzyme Inhibitors and/or Beta Blockers on the Cardiomyopathy in Duchenne Muscular Dystrophy
Viollet, Laurence, PhD, Thrush, Philip T., MD, Flanigan, Kevin M., MD, Mendell, Jerry R., MD, Allen, Hugh D., MD
Published in The American journal of cardiology (01.07.2012)
Published in The American journal of cardiology (01.07.2012)
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Journal Article
Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the mdx Mouse Model of Duchenne Muscular Dystrophy
Potter, Rachael A, Griffin, Danielle A, Heller, Kristin N, Peterson, Ellyn L, Clark, Emma K, Mendell, Jerry R, Rodino-Klapac, Louise R
Published in Human gene therapy (01.04.2021)
Published in Human gene therapy (01.04.2021)
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Journal Article
Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes
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
Published in Pediatrics (Evanston) (01.09.2020)
Published in Pediatrics (Evanston) (01.09.2020)
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Journal Article
AAV1.NT-3 gene therapy for X-linked Charcot-Marie-Tooth neuropathy type 1
Ozes, Burcak, Myers, Morgan, Moss, Kyle, Mckinney, Jennifer, Ridgley, Alicia, Chen, Lei, Bai, Shasha, Abrams, Charles K, Freidin, Mona M, Mendell, Jerry R, Sahenk, Zarife
Published in Gene therapy (01.04.2022)
Published in Gene therapy (01.04.2022)
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Journal Article
Astrocytes from familial and sporadic ALS patients are toxic to motor neurons
Kaspar, Brian K, Haidet-Phillips, Amanda M, Hester, Mark E, Miranda, Carlos J, Meyer, Kathrin, Braun, Lyndsey, Frakes, Ashley, Song, SungWon, Likhite, Shibi, Murtha, Matthew J, Foust, Kevin D, Rao, Meghan, Eagle, Amy, Kammesheidt, Anja, Christensen, Ashley, Mendell, Jerry R, Burghes, Arthur H M
Published in Nature biotechnology (10.08.2011)
Published in Nature biotechnology (10.08.2011)
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Journal Article
Clinical development on the frontier: gene therapy for duchenne muscular dystrophy
Asher, Damon R., Thapa, Khampaseuth, Dharia, Sachi D., Khan, Navid, Potter, Rachael A., Rodino-Klapac, Louise R., Mendell, Jerry R.
Published in Expert opinion on biological therapy (03.03.2020)
Published in Expert opinion on biological therapy (03.03.2020)
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Journal Article
Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy
Griggs, Robert C, Miller, J Phillip, Greenberg, Cheryl R, Fehlings, Darcy L, Pestronk, Alan, Mendell, Jerry R, Moxley, 3rd, Richard T, King, Wendy, Kissel, John T, Cwik, Valerie, Vanasse, Michel, Florence, Julaine M, Pandya, Shree, Dubow, Jordan S, Meyer, James M
Published in Neurology (15.11.2016)
Published in Neurology (15.11.2016)
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Journal Article
A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy
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
Published in Molecular therapy (01.01.2015)
Published in Molecular therapy (01.01.2015)
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Journal Article
Reducing sarcolipin expression mitigates Duchenne muscular dystrophy and associated cardiomyopathy in mice
Voit, Antanina, Patel, Vishwendra, Pachon, Ronald, Shah, Vikas, Bakhutma, Mohammad, Kohlbrenner, Erik, McArdle, Joseph J, Dell'Italia, Louis J, Mendell, Jerry R, Xie, Lai-Hua, Hajjar, Roger J, Duan, Dongsheng, Fraidenraich, Diego, Babu, Gopal J
Published in Nature communications (20.10.2017)
Published in Nature communications (20.10.2017)
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Journal Article
Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes
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.
Published in Molecular therapy (05.04.2017)
Published in Molecular therapy (05.04.2017)
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Journal Article
LTBP4 genotype predicts age of ambulatory loss in duchenne muscular dystrophy
Flanigan, Kevin M., Ceco, Ermelinda, Lamar, Kay-Marie, Kaminoh, Yuuki, Dunn, Diane M., Mendell, Jerry R., King, Wendy M., Pestronk, Alan, Florence, Julaine M., Mathews, Katherine D., Finkel, Richard S., Swoboda, Kathryn J., Gappmaier, Eduard, Howard, Michael T., Day, John W., McDonald, Craig, McNally, Elizabeth M., Weiss, Robert B.
Published in Annals of neurology (01.04.2013)
Published in Annals of neurology (01.04.2013)
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Journal Article
Validity of remote live stream video evaluation of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy
P Lowes, Linda, Alfano, Lindsay N, Iammarino, Megan A, Reash, Natalie F, Giblin, Kathryn, Hu, Larry, Yu, Lixi, Wang, Shufang, Salazar, Rachel, Mendell, Jerry R
Published in PloS one (16.05.2024)
Published in PloS one (16.05.2024)
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