Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study von Finkel, Richard S, Dr, Chiriboga, Claudia A, MD, Vajsar, Jiri, MD, Day, John W, MD, Montes, Jacqueline, EdD, De Vivo, Darryl C, MD, Yamashita, Mason, MD, Rigo, Frank, PhD, Hung, Gene, MD, Schneider, Eugene, MD, Norris, Daniel A, PhD, Xia, Shuting, MS, Bennett, C Frank, PhD, Bishop, Kathie M, PhD

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Observational study of spinal muscular atrophy type I and implications for clinical trials von Finkel, Richard S, McDermott, Michael P, Kaufmann, Petra, Darras, Basil T, Chung, Wendy K, Sproule, Douglas M, Kang, Peter B, Foley, A Reghan, Yang, Michelle L, Martens, William B, Oskoui, Maryam, Glanzman, Allan M, Flickinger, Jean, Montes, Jacqueline, Dunaway, Sally, OʼHagen, Jessica, Quigley, Janet, Riley, Susan, Benton, Maryjane, Ryan, Patricia A, Montgomery, Megan, Marra, Jonathan, Gooch, Clifton, De Vivo, Darryl C

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Revised upper limb module for spinal muscular atrophy: Development of a new module von Mazzone, Elena S., Mayhew, Anna, Montes, Jacqueline, Ramsey, Danielle, Fanelli, Lavinia, Young, Sally Dunaway, Salazar, Rachel, De Sanctis, Roberto, Pasternak, Amy, Glanzman, Allan, Coratti, Giorgia, Civitello, Matthew, Forcina, Nicola, Gee, Richard, Duong, Tina, Pane, Marika, Scoto, Mariacristina, Pera, Maria Carmela, Messina, Sonia, Tennekoon, Gihan, Day, John W., Darras, Basil T., Vivo, Darryl C., Finkel, Richard, Muntoni, Francesco, Mercuri, Eugenio

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Physical exercise training for type 3 spinal muscular atrophy von Bartels, Bart, Montes, Jacqueline, van der Pol, W Ludo, de Groot, Janke F, Bartels, Bart

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A post pandemic roadmap toward remote assessment for neuromuscular disorders: limitations and opportunities von Montes, Jacqueline, Eichinger, Katy J, Pasternak, Amy, Yochai, Cara, Krosschell, Kristin J

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Longitudinal natural history of type I spinal muscular atrophy: a critical review von Mercuri, Eugenio, Lucibello, Simona, Perulli, Marco, Coratti, Giorgia, de Sanctis, Roberto, Pera, Maria Carmela, Pane, Marika, Montes, Jacqueline, de Vivo, Darryl C., Darras, Basil T., Kolb, Stephen J., Finkel, Richard S.

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Ambulatory function in spinal muscular atrophy: Age-related patterns of progression von Montes, Jacqueline, McDermott, Michael P, Mirek, Elizabeth, Mazzone, Elena S, Main, Marion, Glanzman, Allan M, Duong, Tina, Young, Sally Dunaway, Salazar, Rachel, Pasternak, Amy, Gee, Richard, De Sanctis, Roberto, Coratti, Giorgia, Forcina, Nicola, Fanelli, Lavinia, Ramsey, Danielle, Milev, Evelin, Civitello, Matthew, Pane, Marika, Pera, Maria Carmela, Scoto, Mariacristina, Day, John W, Tennekoon, Gihan, Finkel, Richard S, Darras, Basil T, Muntoni, Francesco, De Vivo, Darryl C, Mercuri, Eugenio

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Essential competencies for physical therapist managing individuals with spinal muscular atrophy: A delphi study von Timmerberg, Jean Fitzpatrick, Krosschell, Kristin J., Young, Sally Dunaway, Uher, David, Yun, Chris, Montes, Jacqueline

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Revised Hammersmith Scale for spinal muscular atrophy: A SMA specific clinical outcome assessment tool von Ramsey, Danielle, Scoto, Mariacristina, Mayhew, Anna, Main, Marion, Mazzone, Elena S, Montes, Jacqueline, de Sanctis, Roberto, Dunaway Young, Sally, Salazar, Rachel, Glanzman, Allan M, Pasternak, Amy, Quigley, Janet, Mirek, Elizabeth, Duong, Tina, Gee, Richard, Civitello, Matthew, Tennekoon, Gihan, Pane, Marika, Pera, Maria Carmela, Bushby, Kate, Day, John, Darras, Basil T, De Vivo, Darryl, Finkel, Richard, Mercuri, Eugenio, Muntoni, Francesco

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Validation of a modified version of the gross motor function measure in PPPR5D related neurodevelopmental disorder von Kanner, Cara H, Uher, David, Zreibe, Kyle, Beard, Gabriella, Patterson, Madison, Harris, Matthew, Doerger, Jerome, Calamia, Sean, Chung, Wendy K, Montes, Jacqueline

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Accurate COP Trajectory Estimation in Healthy and Pathological Gait Using Multimodal Instrumented Insoles and Deep Learning Models von Duong, Ton T. H., Uher, David, Young, Sally Dunaway, Farooquee, Rabia, Druffner, Abigail, Pasternak, Amy, Kanner, Cara, Fragala-Pinkham, Maria, Montes, Jacqueline, Zanotto, Damiano

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Long-Term Clinical Course of Glut1 Deficiency Syndrome von Alter, Aliza S., Engelstad, Kristin, Hinton, Veronica J., Montes, Jacqueline, Pearson, Toni S., Akman, Cigdem I., De Vivo, Darryl C.

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Brazilian version of the Hammersmith Functional Motor Scale Expanded: cross-cultural adaptation and validation von Moura, Ana Carolina Monteiro Lessa de, Carvalhais, Marina Belisário, Sampaio, Gabriela Palhares Campolina, Pinhati, Clara Catharino, Montes, Jacqueline, Gurgel-Giannetti, Juliana

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Phase II trial of CoQ10 for ALS finds insufficient evidence to justify phase III von Kaufmann, Petra, Thompson, John L.P., Levy, Gilberto, Buchsbaum, Richard, Shefner, Jeremy, Krivickas, Lisa S., Katz, Jonathan, Rollins, Yvonne, Barohn, Richard J., Jackson, Carlayne E., Tiryaki, Ezgi, Lomen-Hoerth, Catherine, Armon, Carmel, Tandan, Rup, Rudnicki, Stacy A., Rezania, Kourosh, Sufit, Robert, Pestronk, Alan, Novella, Steven P., Heiman-Patterson, Terry, Kasarskis, Edward J., Pioro, Erik P., Montes, Jacqueline, Arbing, Rachel, Vecchio, Darleen, Barsdorf, Alexandra, Mitsumoto, Hiroshi, Levin, Bruce

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Limitations of 6‐minute walk test reference values for spinal muscular atrophy von Goodwin, Ashley M., Cornett, Kayla M.D., McKay, Marnee J., Burns, Joshua, Garber, Carol Ewing, De Vivo, Darryl C., Montes, Jacqueline

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Nusinersen in pediatric and adult patients with type III spinal muscular atrophy von Pera, Maria Carmela, Coratti, Giorgia, Bovis, Francesca, Pane, Marika, Pasternak, Amy, Montes, Jacqueline, Sansone, Valeria A., Dunaway Young, Sally, Duong, Tina, Messina, Sonia, Mizzoni, Irene, D’Amico, Adele, Civitello, Matthew, Glanzman, Allan M., Bruno, Claudio, Salmin, Francesca, Morando, Simone, De Sanctis, Roberto, Sframeli, Maria, Antonaci, Laura, Frongia, Anna Lia, Rohwer, Annemarie, Scoto, Mariacristina, De Vivo, Darryl C., Darras, Basil T., Day, John, Martens, William, Patanella, Katia A., Bertini, Enrico, Muntoni, Francesco, Finkel, Richard, Mercuri, Eugenio

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Scientific rationale for a higher dose of nusinersen von Finkel, Richard S., Ryan, Monique M., Pascual Pascual, Samuel Ignacio, Day, John W., Mercuri, Eugenio, De Vivo, Darryl C., Foster, Richard, Montes, Jacqueline, Gurgel‐Giannetti, Juliana, MacCannell, Drew, Berger, Zdenek

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Quantitative gait assessment in children with 16p11.2 syndrome von Goldman, Sylvie, McCullough, Aston K, Young, Sally Dunaway, Mueller, Carly, Stahl, Adrianna, Zoeller, Audrey, Abbruzzese, Laurel Daniels, Rao, Ashwini K, Montes, Jacqueline

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Validation of the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) von Glanzman, Allan M, McDermott, Michael P, Montes, Jacqueline, Martens, William B, Flickinger, Jean, Riley, Susan, Quigley, Janet, Dunaway, Sally, O'Hagen, Jessica, Deng, Liyong, Chung, Wendy K, Tawil, Rabi, Darras, Basil T, Yang, Michele, Sproule, Douglas, De Vivo, Darryl C, Kaufmann, Petra, Finkel, Richard S

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What system controls balance in children with charcot‐marie‐tooth disease? von Yoon, Lisa, Montes, Jacqueline

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