Vision Loss and Symmetric Basal Ganglia Lesions in Leber Hereditary Optic Neuropathy von Mercuri, Matthew A, White, Halina, Oliveira, Cristiano

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Construing stigmatized identities: A golden section study von Raskin, Jonathan D., Harasym, Melanie T., Mercuri, Matthew A., Widrick, Rebekah M.

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Abstract 140: Association Between Epilepsy and the Risk of Ischemic Stroke or Myocardial Infarction von Mercuri, Matthew A, Merkler, Alexander E, Parikh, Neal S, Reznik, Michael E, Kamel, Hooman

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Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy von Mendell, Jerry R., Goemans, Nathalie, Lowes, Linda P., Alfano, Lindsay N., Berry, Katherine, Shao, James, Kaye, Edward M., Mercuri, Eugenio

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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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Patient "No-Show" Increases the Risk of 90-Day Complications Following Primary Total Knee Arthroplasty: A Retrospective Cohort Study of 6,776 Patients von Mun, Jeffrey S, Parry, Matthew W, Tang, Alex, Manikowski, Jesse J, Crinella, Cory, Mercuri, John J

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Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy von Straub, Volker, MD, Balabanov, Pavel, PhD, Bushby, Kate, MD, Ensini, Monica, PhD, Goemans, Nathalie, MD, De Luca, Annamaria, PhD, Pereda, Alejandra, Hemmings, Robert, Campion, Giles, MD, Kaye, Edward, MD, Arechavala-Gomeza, Virginia, PhD, Goyenvalle, Aurelie, PhD, Niks, Erik, MD, Veldhuizen, Olav, Furlong, Pat, Stoyanova-Beninska, Violeta, MD, Wood, Matthew J, PhD, Johnson, Alex, Mercuri, Eugenio, MD, Muntoni, Francesco, MD, Sepodes, Bruno, PhD, Haas, Manuel, PharmD, Vroom, Elizabeth, DMD, Aartsma-Rus, Annemieke, Dr

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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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Physician Perspectives on Telemedicine at an Integrated Health System During the COVID-19 Pandemic von Luciani, A Michael, Parry, Matthew, Udoeyo, Idorenyin F, Suk, Michael, Mercuri, John J, DelSole, Edward M

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Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy von Finkel, Richard S, Mercuri, Eugenio, Darras, Basil T, Connolly, Anne M, Kuntz, Nancy L, Kirschner, Janbernd, Chiriboga, Claudia A, Saito, Kayoko, Servais, Laurent, Tizzano, Eduardo, Topaloglu, Haluk, Tulinius, Már, Montes, Jacqueline, Glanzman, Allan M, Bishop, Kathie, Zhong, Z. John, Gheuens, Sarah, Bennett, C. Frank, Schneider, Eugene, Farwell, Wildon, De Vivo, Darryl C

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Loss-of-Function Mutations in APPL1 in Familial Diabetes Mellitus von Prudente, Sabrina, Jungtrakoon, Prapaporn, Marucci, Antonella, Ludovico, Ornella, Buranasupkajorn, Patinut, Mazza, Tommaso, Hastings, Timothy, Milano, Teresa, Morini, Eleonora, Mercuri, Luana, Bailetti, Diego, Mendonca, Christine, Alberico, Federica, Basile, Giorgio, Romani, Marta, Miccinilli, Elide, Pizzuti, Antonio, Carella, Massimo, Barbetti, Fabrizio, Pascarella, Stefano, Marchetti, Piero, Trischitta, Vincenzo, Di Paola, Rosa, Doria, Alessandro

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Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for diagnosis, rehabilitation, orthopedic and nutritional care von Mercuri, Eugenio, Finkel, Richard S., Muntoni, Francesco, Wirth, Brunhilde, Montes, Jacqueline, Main, Marion, Mazzone, Elena S., Vitale, Michael, Snyder, Brian, Quijano-Roy, Susana, Bertini, Enrico, Davis, Rebecca Hurst, Meyer, Oscar H., Simonds, Anita K., Schroth, Mary K., Graham, Robert J., Kirschner, Janbernd, Iannaccone, Susan T., Crawford, Thomas O., Woods, Simon, Qian, Ying, Sejersen, Thomas, Muntoni, Francesco, Wirth, Brunhilde, Tiziano, Francesco Danilo, Kirschner, Janbernd, Tizzano, Eduardo, Topaloglu, Haluk, Swoboda, Kathy, Laing, Nigel, Kayoko, Saito, Prior, Thomas, Chung, Wendy K., Wu, Shou-Mei, Montes, Jacqueline, Mazzone, Elena, Main, Marion, Coleman, Caron, Gee, Richard, Glanzman, Allan, Kroksmark, Anna-Karin, Krosschell, Kristin, Nelson, Leslie, Rose, Kristy, Stępień, Agnieszka, Vuillerot, Carole, Vitale, Michael, Snyder, Brian, Quijano-Roy, Susana, Dubousset, Jean, Farrington, David, Flynn, Jack, Halanski, Matthew, Hasler, Carol, Miladi, Lotfi, Reilly, Christopher, Roye, Benjamin, Sponseller, Paul, Yazici, Muharrem, Hurst, Rebecca, Bertini, Enrico, Tarrant, Stacey, Barja, Salesa, Bertoli, Simona, Crawford, Thomas, Foust, Kevin, Kyle, Barbara, Rodan, Lance, Roper, Helen, Seffrood, Erin, Swoboda, Kathryn, Szlagatys-Sidorkiewicz, Agnieszka

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The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy von Scaglioni, Dominic, Catapano, Francesco, Ellis, Matthew, Torelli, Silvia, Chambers, Darren, Feng, Lucy, Beck, Matthew, Sewry, Caroline, Monforte, Mauro, Harriman, Shawn, Koenig, Erica, Malhotra, Jyoti, Popplewell, Linda, Guglieri, Michela, Straub, Volker, Mercuri, Eugenio, Servais, Laurent, Phadke, Rahul, Morgan, Jennifer, Muntoni, Francesco

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Vision Loss with Symmetric Putaminal Lesions in Leber’s Hereditary Optic Neuropathy (P5.194) von Mercuri, Matthew, White, Halina, Oliveira, Cristiano

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Long-Term Follow-Up (LTFU) of Onasemnogene Abeparvovec Gene Therapy in Spinal Muscular Atrophy (SMA) (2301) von Mendell, Jerry, Finkel, Richard, Mercuri, Eugenio, Strauss, Kevin, Day, John, Kleyn, Aaron, Chand, Deepa, Tauscher-Wisniewski, Sitra, Meriggioli, Matthew

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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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IQ After Pediatric Concussion von Ware, Ashley L, McLarnon, Matthew J W, Lapointe, Andrew P, Brooks, Brian L, Bacevice, Ann, Bangert, Barbara A, Beauchamp, Miriam H, Bigler, Erin D, Bjornson, Bruce, Cohen, Daniel M, Craig, William, Doan, Quynh, Freedman, Stephen B, Goodyear, Bradley G, Gravel, Jocelyn, Mihalov, H Leslie K, Minich, Nori Mercuri, Taylor, H Gerry, Zemek, Roger, Yeates, Keith Owen

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Novel mutations expand the clinical spectrum of DYNC1H1-associated spinal muscular atrophy von Scoto, Mariacristina, Rossor, Alexander M, Harms, Matthew B, Cirak, Sebahattin, Calissano, Mattia, Robb, Stephanie, Manzur, Adnan Y, Martínez Arroyo, Amaia, Rodriguez Sanz, Aida, Mansour, Sahar, Fallon, Penny, Hadjikoumi, Irene, Klein, Andrea, Yang, Michele, De Visser, Marianne, Overweg-Plandsoen, W.C.G (Truus), Baas, Frank, Taylor, J Paul, Benatar, Michael, Connolly, Anne M, Al-Lozi, Muhammad T, Nixon, John, de Goede, Christian G.E.L, Foley, A Reghan, Mcwilliam, Catherine, Pitt, Matthew, Sewry, Caroline, Phadke, Rahul, Hafezparast, Majid, Chong, W.K “Kling, Mercuri, Eugenio, Baloh, Robert H, Reilly, Mary M, Muntoni, Francesco

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Prognostic indicators and outcomes of hospitalised COVID-19 patients with neurological disease: An individual patient data meta-analysis von Singh, Bhagteshwar, Lant, Suzannah, Cividini, Sofia, Cattrall, Jonathan W S, Goodwin, Lynsey C, Benjamin, Laura, Michael, Benedict D, Khawaja, Ayaz, Matos, Aline de Moura Brasil, Alkeridy, Walid, Pilotto, Andrea, Lahiri, Durjoy, Rawlinson, Rebecca, Mhlanga, Sithembinkosi, Lopez, Evelyn C, Sargent, Brendan F, Somasundaran, Anushri, Tamborska, Arina, Webb, Glynn, Younas, Komal, Al Sami, Yaqub, Babu, Heavenna, Banks, Tristan, Cavallieri, Francesco, Cohen, Matthew, Davies, Emma, Dhar, Shalley, Fajardo Modol, Anna, Farooq, Hamzah, Harte, Jeffrey, Hey, Samuel, Joseph, Albert, Karthikappallil, Dileep, Kassahun, Daniel, Lipunga, Gareth, Mason, Rachel, Minton, Thomas, Mond, Gabrielle, Poxon, Joseph, Rabas, Sophie, Soothill, Germander, Zedde, Marialuisa, Yenkoyan, Konstantin, Brew, Bruce, Contini, Erika, Cysique, Lucette, Zhang, Xin, Maggi, Pietro, van Pesch, Vincent, Lechien, Jérome, Saussez, Sven, Heyse, Alex, Brito Ferreira, Maria Lúcia, Soares, Cristiane N, Elicer, Isabel, Eugenín-von Bernhardi, Laura, Ñancupil Reyes, Waleng, Yin, Rong, Azab, Mohammed A, Abd-Allah, Foad, Elkady, Ahmed, Escalard, Simon, Corvol, Jean-Christophe, Delorme, Cécile, Tattevin, Pierre, Bigaut, Kévin, Lorenz, Norbert, Hornuss, Daniel, Hosp, Jonas, Rieg, Siegbert, Wagner, Dirk, Knier, Benjamin, Lingor, Paul, Winkler, Andrea Sylvia, Sharifi-Razavi, Athena, Moein, Shima T, SeyedAlinaghi, SeyedAhmad, JamaliMoghadamSiahkali, Saeidreza, Morassi, Mauro, Padovani, Alessandro, Giunta, Marcello, Libri, Ilenia, Beretta, Simone, Ravaglia, Sabrina, Foschi, Matteo, Calabresi, Paolo, Primiano, Guido, Servidei, Serenella, Biagio Mercuri, Nicola, Liguori, Claudio, Pierantozzi, Mariangela, Sarmati, Loredana, Boso, Federica, Garazzino, Silvia, Mariotto, Sara, Patrick, Kimani N, Costache, Oana, Pincherle, Alexander, Klok, Frederikus A, Meza, Roger

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A critical review of patient and parent caregiver oriented tools to assess health-related quality of life, activity of daily living and caregiver burden in spinal muscular atrophy von Messina, Sonia, Frongia, Anna Lia, Antonaci, Laura, Pera, Maria Carmela, Coratti, Giorgia, Pane, Marika, Pasternak, Amy, Civitello, Matthew, Montes, Jacqueline, Mayhew, Anna, Finkel, Richard, Muntoni, Francesco, Mercuri, Eugenio

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