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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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
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
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
Feasibility and utility of in-home body weight support harness system use in young children treated for spinal muscular atrophy: A single-arm prospective cohort study
Iammarino, Megan A, Alfano, Lindsay N, Reash, Natalie F, Sabo, Brenna, Conroy, Sara, Noritz, Garey, Wendland, Madalynn, Lowes, Linda P
Published in PloS one (19.03.2024)
Published in PloS one (19.03.2024)
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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
Development of Duchenne Video Assessment scorecards to evaluate ease of movement among those with Duchenne muscular dystrophy
Contesse, Marielle G, Lowes, Linda P, White, Michelle K, Dalle Pazze, Laura, McSherry, Christine, Alfano, Lindsay N, Iammarino, Megan, Reash, Natalie, Bonarrigo, Kelly, Kiefer, Michael, Laubscher, Katie, McIntyre, Melissa, Mockler, Shelley, Nelson, Leslie, Vogel, Leslie, Leffler, Mindy G
Published in PloS one (13.04.2022)
Published in PloS one (13.04.2022)
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Journal Article
First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Development in Neuromuscular Diseases
Servais, Laurent, Camino, Eric, Clement, Aude, McDonald, Craig M., Lukawy, Jacek, Lowes, Linda P., Eggenspieler, Damien, Cerreta, Francesca, Strijbos, Paul
Published in Digital biomarkers (01.05.2021)
Published in Digital biomarkers (01.05.2021)
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Remote Delivery of Motor Function Assessment and Training for Clinical Trials in Neuromuscular Disease: A Response to the COVID-19 Global Pandemic
James, Meredith K., Rose, Kristy, Alfano, Lindsay N., Reash, Natalie F., Eagle, Michelle, Lowes, Linda P.
Published in Frontiers in genetics (29.10.2021)
Published in Frontiers in genetics (29.10.2021)
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Journal Article
Home‐based video assessment of ease of movement for patients with Duchenne: Interviews with physical therapists to select movement tasks
Contesse, Marielle G., Hodges, Jason, Staunton, Hannah, Lowes, Linda P., Elmankabadi, Bassem, Elder, Sonya J., Ormazabal, Maitea Guridi, Dalle Pazze, Laura, Leffler, Mindy G.
Published in Physiotherapy research international : the journal for researchers and clinicians in physical therapy (01.07.2023)
Published in Physiotherapy research international : the journal for researchers and clinicians in physical therapy (01.07.2023)
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Journal Article
Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy
Mendell, Jerry R, Shieh, Perry B, McDonald, Craig M, Sahenk, Zarife, Lehman, Kelly J, Lowes, Linda P, Reash, Natalie F, Iammarino, Megan A, Alfano, Lindsay N, Sabo, Brenna, Woods, Jeremy D, Skura, Christy L, Mao, Howard C, Staudt, Loretta A, Griffin, Danielle A, Lewis, Sarah, Wang, Shufang, Potter, Rachael A, Singh, Teji, Rodino-Klapac, Louise R
Published in Frontiers in cell and developmental biology (11.07.2023)
Published in Frontiers in cell and developmental biology (11.07.2023)
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Journal Article
Modeling functional decline over time in sporadic inclusion body myositis
Alfano, Lindsay N., Yin, Han, Dvorchik, Igor, Maus, Elizabeth G., Flanigan, Kevin M., Mendell, Jerry R., Lowes, Linda P.
Published in Muscle & nerve (01.04.2017)
Published in Muscle & nerve (01.04.2017)
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Journal Article
Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases
Duong, Tina, Krosschell, Kristin J, James, Meredith K, Nelson, Leslie, Alfano, Lindsay N, Eichinger, Katy, Mazzone, Elena, Rose, Kristy, Lowes, Linda P, Mayhew, Anna, Florence, Julaine, King, Wendy, Senesac, Claudia R, Eagle, Michelle
Published in Frontiers in genetics (10.11.2021)
Published in Frontiers in genetics (10.11.2021)
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Journal Article
WiTNNess : An international natural history study of infantile‐onset TNNT1 myopathy
Strauss, Kevin A., Carson, Vincent J., Bolettieri, Emilienne, Everett, Mariah, Bollinger, Ashton, Bowser, Lauren E., Beiler, Keturah, Young, Millie, Edvardson, Simon, Fraenkel, Nitay, D'Amico, Adele, Bertini, Enrico, Lingappa, Lokesh, Chowdhury, Devyani, Lowes, Linda P., Iammarino, Megan, Alfano, Lindsay N., Brigatti, Karlla W.
Published in Annals of clinical and translational neurology (01.11.2023)
Published in Annals of clinical and translational neurology (01.11.2023)
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Journal Article
Correlation of knee strength to functional outcomes in becker muscular dystrophy
Alfano, Lindsay N., Lowes, Linda P., Flanigan, Kevin M., Mendell, Jerry R.
Published in Muscle & nerve (01.04.2013)
Published in Muscle & nerve (01.04.2013)
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Journal Article
Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy
Mendell, Jerry R, Al-Zaidy, Samiah A, Lehman, Kelly J, McColly, Markus, Lowes, Linda P, Alfano, Lindsay N, Reash, Natalie F, Iammarino, Megan A, Church, Kathleen R, Kleyn, Aaron, Meriggioli, Matthew N, Shell, Richard
Published in Archives of neurology (Chicago) (01.07.2021)
Published in Archives of neurology (Chicago) (01.07.2021)
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Journal Article
Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial
Mendell, Jerry R, Sahenk, Zarife, Lehman, Kelly, Nease, Carrie, Lowes, Linda P, Miller, Natalie F, Iammarino, Megan A, Alfano, Lindsay N, Nicholl, Amanda, Al-Zaidy, Samiah, Lewis, Sarah, Church, Kathleen, Shell, Richard, Cripe, Linda H, Potter, Rachael A, Griffin, Danielle A, Pozsgai, Eric, Dugar, Ashish, Hogan, Mark, Rodino-Klapac, Louise R
Published in JAMA neurology (01.09.2020)
Published in JAMA neurology (01.09.2020)
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Journal Article
Emerging therapeutic options for sporadic inclusion body myositis
Alfano, Lindsay N, Lowes, Linda P
Published in Therapeutics and clinical risk management (01.01.2015)
Published in Therapeutics and clinical risk management (01.01.2015)
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Journal Article
Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy
Lowes, Linda P., Alfano, Lindsay N., Arnold, W. David, Shell, Richard, Prior, Thomas W., McColly, Markus, Lehman, Kelly J., Church, Kathleen, Sproule, Douglas M., Nagendran, Sukumar, Menier, Melissa, Feltner, Douglas E., Wells, Courtney, Kissel, John T., Al-Zaidy, Samiah, Mendell, Jerry
Published in Pediatric neurology (01.09.2019)
Published in Pediatric neurology (01.09.2019)
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