Search Results - "Iammarino, Megan"

Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy by 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

“…This ongoing study assesses long-term safety and durability of response in infants with spinal muscular atrophy (SMA) type 1 after dosing with onasemnogene…”
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Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial by 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

“…Micro-dystrophin gene transfer shows promise for treating patients with Duchenne muscular dystrophy (DMD) using recombinant adeno-associated virus serotype…”
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Validity of remote live stream video evaluation of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy by 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

“…Conducting functional assessments remotely can help alleviate the burden of in-person assessment on patients with Duchenne muscular dystrophy and their…”
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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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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 by Iammarino, Megan A, Alfano, Lindsay N, Reash, Natalie F, Sabo, Brenna, Conroy, Sara, Noritz, Garey, Wendland, Madalynn, Lowes, Linda P

“…This single-arm prospective cohort study aimed to evaluate the feasibility and utility of in-home body weight support harness system (BWSS) use in children…”
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Long‐term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial by Mendell, Jerry R., Sahenk, Zarife, Lehman, Kelly J., Lowes, Linda P., Reash, Natalie F., Iammarino, Megan A., Alfano, Lindsay N., Lewis, Sarah, Church, Kathleen, Shell, Richard, Potter, Rachael A., Griffin, Danielle A., Hogan, Mark, Wang, Shufang, Mason, Stefanie, Darton, Eddie, Rodino‐Klapac, Louise R.

“…Introduction/Aims Delandistrogene moxeparvovec is indicated in the United States for the treatment of ambulatory pediatric patients aged 4 through 5 years with…”
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Ambient floor vibration sensing advances the accessibility of functional gait assessments for children with muscular dystrophies by Dong, Yiwen, Iammarino, Megan, Liu, Jingxiao, Codling, Jesse, Fagert, Jonathon, Mirshekari, Mostafa, Lowes, Linda, Zhang, Pei, Noh, Hae Young

“…Muscular dystrophies (MD) are a group of genetic neuromuscular disorders that cause progressive weakness and loss of muscles over time, influencing 1 in…”
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Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results by Mendell, Jerry R., Pozsgai, Eric R., Lewis, Sarah, Griffin, Danielle A., Lowes, Linda P., Alfano, Lindsay N., Lehman, Kelly J., Church, Kathleen, Reash, Natalie F., Iammarino, Megan A., Sabo, Brenna, Potter, Rachael, Neuhaus, Sarah, Li, Xiaoxi, Stevenson, Herb, Rodino-Klapac, Louise R.

“…Limb-girdle muscular dystrophy 2E/R4 is caused by mutations in the β-sarcoglycan ( SGCB ) gene, leading to SGCB deficiency and consequent muscle loss. We…”
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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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Development of Duchenne Video Assessment scorecards to evaluate ease of movement among those with Duchenne muscular dystrophy by 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

“…Patients with Duchenne muscular dystrophy (DMD) adopt compensatory movement patterns as muscles weaken. The Duchenne Video Assessment (DVA) measures patient…”
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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 by 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

“…Delandistrogene moxeparvovec (SRP-9001) is an investigational gene transfer therapy designed for targeted expression of SRP-9001 dystrophin protein, a…”
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ACTIVE (Ability Captured Through Interactive Video Evaluation) workspace volume video game to quantify meaningful change in spinal muscular atrophy by Alfano, Lindsay N, Miller, Natalie F, Iammarino, Megan A, Moore Clingenpeel, Melissa, Lowes, Suzanne L, Dugan, Margaret E, Kissel, John T, Al Zaidy, Samiah, Tsao, Chang‐Yong, Lowes, Linda P

“…Aim To evaluate the utility of Ability Captured Through Interactive Video Evaluation (ACTIVE) scaled scores to quantify meaningful change in individuals with…”
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Comparison of strength testing modalities in dysferlinopathy by Reash, Natalie F., James, Meredith K., Alfano, Lindsay N., Mayhew, Anna G., Jacobs, Marni, Iammarino, Megan A., Holsten, Scott, Sakamoto, Chikako, Tateishi, Takayuki, Yajima, Hiroyuki, Duong, Tina, Wolf, Brittney, Gee, Richard, Bharucha‐Goebel, Diana X., Bravver, Elena, Mori‐Yoshimura, Madoka, Bushby, Kate, Rufibach, Laura E., Straub, Volker, Lowes, Linda P.

“…Introduction/Aims Dysferlinopathy demonstrates heterogeneity in muscle weakness between patients, which can progress at different rates over time. Changing…”
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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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Increase in Full-Length Dystrophin by Exon Skipping in Duchenne Muscular Dystrophy Patients with Single Exon Duplications: An Open-label Study by Nicolau, Stefan, Malhotra, Jyoti, Kaler, Maryann, Magistrado-Coxen, Pamela, Iammarino, Megan A, Reash, Natalie F, Frair, Emma C, Wijeratne, Saranga, Kelly, Benjamin J, White, Peter, Lowes, Linda P, Waldrop, Megan A, Flanigan, Kevin M

“…Single exon duplications account for disease in a minority of Duchenne muscular dystrophy patients. Exon skipping in these patients has the potential to be…”
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A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2 by Flanigan, Kevin M., Vetter, Tatyana A., Simmons, Tabatha R., Iammarino, Megan, Frair, Emma C., Rinaldi, Federica, Chicoine, Louis G., Harris, Johan, Cheatham, John P., Cheatham, Sharon L., Boe, Brian, Waldrop, Megan A., Zygmunt, Deborah A., Packer, Davin, Martin, Paul T.

“…In a phase 1/2, open-label dose escalation trial, we delivered rAAVrh74.MCK.GALGT2 (also B4GALNT2) bilaterally to the legs of two boys with Duchenne muscular…”
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WiTNNess : An international natural history study of infantile‐onset TNNT1 myopathy by 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.

“…ABSTRACT Objective We created WiTNNess as a hybrid prospective/cross‐sectional observational study to simulate a clinical trial for infantile‐onset TNNT1…”
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P314: Long-term safety and sustained functional benefit in patients with DMD 4 years post-treatment with delandistrogene moxeparvovec: A phase 1/2a study by O’Rourke, Erin, Mendell, Jerry R., Sahenk, Zarife, Lehman, Kelly J., Lowes, Linda P., Reash, Natalie F., Iammarino, Megan, Alfano, Lindsay, Lewis, Sarah, Church, Kathleen, Shell, Richard, Potter, Rachael A., Griffin, Danielle, Hogan, Mark J., Wang, Shufang, Mason, Stefanie, Darton, Eddie, Rodino-Klapac, Louise

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Validation of the North Star Assessment for Limb-Girdle Type Muscular Dystrophies by James, Meredith K, Alfano, Lindsay N, Muni-Lofra, Robert, Reash, Natalie F, Sodhi, Jassi, Iammarino, Megan A, Moat, Dionne, Shannon, Kianna, McCallum, Michelle, Richardson, Mark, Eagle, Michelle, Straub, Volker, Marini-Bettolo, Chiara, Lowes, Linda P, Mayhew, Anna G

“…Abstract Objective The North Star Assessment for limb-girdle type muscular dystrophies (NSAD), a clinician-reported outcome measure (ClinRO) of motor…”
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Activity monitoring in neuromuscular disease: Successes, challenges, and a path forward (P5.6-016) by Lowes, Linda, Miller, Natalie, Iammarino, Megan, Dugan, Margaret, Alfano, Lindsay

“…Abstract only…”
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