Overturning the Paradigm of Spinal Muscular Atrophy as Just a Motor Neuron Disease von Yeo, Crystal Jing Jing, Darras, Basil T.

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Proceedings of ASEAN-Australian Engineering Congress (AAEC2022): Engineering Solutions in the Age of Digital Disruption von Choo, Chung Siung, Wong, Basil T, Sharkawi, Khairul Hafiz Bin, Kong, Daniel

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Risdiplam in Type 1 Spinal Muscular Atrophy von Baranello, Giovanni, Darras, Basil T, Day, John W, Deconinck, Nicolas, Klein, Andrea, Masson, Riccardo, Mercuri, Eugenio, Rose, Kristy, El-Khairi, Muna, Gerber, Marianne, Gorni, Ksenija, Khwaja, Omar, Kletzl, Heidemarie, Scalco, Renata S, Seabrook, Timothy, Fontoura, Paulo, Servais, Laurent

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Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls von Darras, Basil T, Masson, Riccardo, Mazurkiewicz-Bełdzińska, Maria, Rose, Kristy, Xiong, Hui, Zanoteli, Edmar, Baranello, Giovanni, Bruno, Claudio, Vlodavets, Dmitry, Wang, Yi, El-Khairi, Muna, Gerber, Marianne, Gorni, Ksenija, Khwaja, Omar, Kletzl, Heidemarie, Scalco, Renata S, Fontoura, Paulo, Servais, Laurent

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Spinal muscular atrophy von Mercuri, Eugenio, Sumner, Charlotte J., Muntoni, Francesco, Darras, Basil T., Finkel, Richard S.

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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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Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy von Chiriboga, Claudia A, Swoboda, Kathryn J, Darras, Basil T, Iannaccone, Susan T, Montes, Jacqueline, De Vivo, Darryl C, Norris, Daniel A, Bennett, C Frank, Bishop, Kathie M

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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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Nusinersen in later-onset spinal muscular atrophy: Long-term results from the phase 1/2 studies von Darras, Basil T, Chiriboga, Claudia A, Iannaccone, Susan T, Swoboda, Kathryn J, Montes, Jacqueline, Mignon, Laurence, Xia, Shuting, Bennett, C Frank, Bishop, Kathie M, Shefner, Jeremy M, Green, Allison M, Sun, Peng, Bhan, Ishir, Gheuens, Sarah, Schneider, Eugene, Farwell, Wildon, De Vivo, Darryl C

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Challenges and opportunities in spinal muscular atrophy therapeutics von Yeo, Crystal J J, Tizzano, Eduardo F, Darras, Basil T

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Systemic nature of spinal muscular atrophy revealed by studying insurance claims von Lipnick, Scott L, Agniel, Denis M, Aggarwal, Rahul, Makhortova, Nina R, Finlayson, Samuel G, Brocato, Alexandra, Palmer, Nathan, Darras, Basil T, Kohane, Isaac, Rubin, Lee L

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Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre,... von Day, John W, Finkel, Richard S, Chiriboga, Claudia A, Connolly, Anne M, Crawford, Thomas O, Darras, Basil T, Iannaccone, Susan T, Kuntz, Nancy L, Peña, Loren D M, Shieh, Perry B, Smith, Edward C, Kwon, Jennifer M, Zaidman, Craig M, Schultz, Meredith, Feltner, Douglas E, Tauscher-Wisniewski, Sitra, Ouyang, Haojun, Chand, Deepa H, Sproule, Douglas M, Macek, Thomas A, Mendell, Jerry R

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Yeo and Darras: Extraneuronal Phenotypes of Spinal Muscular Atrophy von Yeo, Crystal Jing Jing, Darras, Basil T.

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Quantitative muscle ultrasound detects disease progression in Duchenne muscular dystrophy von Zaidman, Craig M., Wu, Jim S., Kapur, Kush, Pasternak, Amy, Madabusi, Lavanya, Yim, Sung, Pacheck, Adam, Szelag, Heather, Harrington, Tim, Darras, Basil T., Rutkove, Seward B.

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An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials von Darras, Basil T., Farrar, Michelle A., Mercuri, Eugenio, Finkel, Richard S., Foster, Richard, Hughes, Steven G., Bhan, Ishir, Farwell, Wildon, Gheuens, Sarah

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Precious SMA natural history data: A benchmark to measure future treatment successes von Darras, Basil T, De Vivo, Darryl C

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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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Ethical Perspectives on Treatment Options with Spinal Muscular Atrophy Patients von Yeo, Crystal J. J., Simmons, Zachary, De Vivo, Darryl C., Darras, Basil T.

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Non-5q spinal muscular atrophies: the alphanumeric soup thickens von Darras, Basil T

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Neurofilament as a potential biomarker for spinal muscular atrophy von Darras, Basil T., Crawford, Thomas O., Finkel, Richard S., Mercuri, Eugenio, De Vivo, Darryl C., Oskoui, Maryam, Tizzano, Eduardo F., Ryan, Monique M., Muntoni, Francesco, Zhao, Guolin, Staropoli, John, McCampbell, Alexander, Petrillo, Marco, Stebbins, Christopher, Fradette, Stephanie, Farwell, Wildon, Sumner, Charlotte J.

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