Risdiplam in Type 1 Spinal Muscular Atrophy
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
Published in The New England journal of medicine (11.03.2021)
Published in The New England journal of medicine (11.03.2021)
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Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls
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
Published in The New England journal of medicine (29.07.2021)
Published in The New England journal of medicine (29.07.2021)
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Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy
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
Published in Neurology (08.03.2016)
Published in Neurology (08.03.2016)
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Systemic nature of spinal muscular atrophy revealed by studying insurance claims
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
Published in PloS one (14.03.2019)
Published in PloS one (14.03.2019)
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Spinal muscular atrophy
Mercuri, Eugenio, Sumner, Charlotte J., Muntoni, Francesco, Darras, Basil T., Finkel, Richard S.
Published in Nature reviews. Disease primers (04.08.2022)
Published in Nature reviews. Disease primers (04.08.2022)
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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, phase 3 trial
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
Published in Lancet neurology (01.04.2021)
Published in Lancet neurology (01.04.2021)
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Nusinersen in later-onset spinal muscular atrophy: Long-term results from the phase 1/2 studies
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
Published in Neurology (21.05.2019)
Published in Neurology (21.05.2019)
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Observational study of spinal muscular atrophy type I and implications for clinical trials
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, O'Hagen, Jessica, Quigley, Janet, Riley, Susan, Benton, Maryjane, Ryan, Patricia A, Montgomery, Megan, Marra, Jonathan, Gooch, Clifton, De Vivo, Darryl C
Published in Neurology (26.08.2014)
Published in Neurology (26.08.2014)
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Revised upper limb module for spinal muscular atrophy: Development of a new module
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
Published in Muscle & nerve (01.06.2017)
Published in Muscle & nerve (01.06.2017)
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Revised Hammersmith Scale for spinal muscular atrophy: A SMA specific clinical outcome assessment tool
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
Published in PloS one (21.02.2017)
Published in PloS one (21.02.2017)
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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
Darras, Basil T., Farrar, Michelle A., Mercuri, Eugenio, Finkel, Richard S., Foster, Richard, Hughes, Steven G., Bhan, Ishir, Farwell, Wildon, Gheuens, Sarah
Published in CNS drugs (01.09.2019)
Published in CNS drugs (01.09.2019)
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Cyber-Physical Systems as an Enabler of Circular Economy to Achieve Sustainable Development Goals: A Comprehensive Review
Ahmed, Aser Alaa, Nazzal, Mohammad A., Darras, Basil M.
Published in International Journal of Precision Engineering and Manufacturing-Green Technology (01.05.2022)
Published in International Journal of Precision Engineering and Manufacturing-Green Technology (01.05.2022)
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