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Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates
Meyer, Kathrin, Ferraiuolo, Laura, Schmelzer, Leah, Braun, Lyndsey, McGovern, Vicki, Likhite, Shibi, Michels, Olivia, Govoni, Alessandra, Fitzgerald, Julie, Morales, Pablo, Foust, Kevin D, Mendell, Jerry R, Burghes, Arthur H M, Kaspar, Brian K
Published in Molecular therapy (01.03.2015)
Published in Molecular therapy (01.03.2015)
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Journal Article
Magnetic resonance reveals mitochondrial dysfunction and muscle remodelling in spinal muscular atrophy
Habets, Laura E, Bartels, Bart, Asselman, Fay-Lynn, Hooijmans, Melissa T, van den Berg, Sandra, Nederveen, Aart J, van der Pol, W Ludo, Jeneson, Jeroen A L
Published in Brain (London, England : 1878) (24.05.2022)
Published in Brain (London, England : 1878) (24.05.2022)
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Journal Article
Is level- and side-specific multifidus asymmetry a marker for lumbar disc pathology?
Battié, Michele C., Niemelainen, Riikka, Gibbons, Laura E., Dhillon, Sukhvinder
Published in The spine journal (01.10.2012)
Published in The spine journal (01.10.2012)
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Journal Article
Effect of Salbutamol on Respiratory Muscle Strength in Spinal Muscular Atrophy
Khirani, Sonia, Dabaj, Ivana, Amaddeo, Alessandro, Olmo Arroyo, Jorge, Ropers, Jacques, Tirolien, Stéphane, Coudert, Véronique, Estournet, Brigitte, Fauroux, Brigitte, Quijano-Roy, Susana
Published in Pediatric neurology (01.08.2017)
Published in Pediatric neurology (01.08.2017)
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Journal Article
Phase II Open Label Study of Valproic Acid in Spinal Muscular Atrophy
Swoboda, Kathryn J., Scott, Charles B., Reyna, Sandra P., Prior, Thomas W., LaSalle, Bernard, Sorenson, Susan L., Wood, Janine, Acsadi, Gyula, Crawford, Thomas O., Kissel, John T., Krosschell, Kristin J., D'Anjou, Guy, Bromberg, Mark B., Schroth, Mary K., Chan, Gary M., Elsheikh, Bakri, Simard, Louise R.
Published in PloS one (14.05.2009)
Published in PloS one (14.05.2009)
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Journal Article
Regional Cervical Cord Atrophy and Disability in Multiple Sclerosis: A Voxel-based Analysis
Valsasina, Paola, Rocca, Maria A., Horsfield, Mark A., Absinta, Martina, Messina, Roberta, Caputo, Domenico, Comi, Giancarlo, Filippi, Massimo
Published in Radiology (01.03.2013)
Published in Radiology (01.03.2013)
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Journal Article
Androgen-dependent impairment of myogenesis in spinal and bulbar muscular atrophy
Malena, Adriana, Pennuto, Maria, Tezze, Caterina, Querin, Giorgia, D’Ascenzo, Carla, Silani, Vincenzo, Cenacchi, Giovanna, Scaramozza, Annarita, Romito, Silvia, Morandi, Lucia, Pegoraro, Elena, Russell, Aaron P., Sorarù, Gianni, Vergani, Lodovica
Published in Acta neuropathologica (01.07.2013)
Published in Acta neuropathologica (01.07.2013)
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Journal Article
MRI of paraspinal muscles in lumbar degenerative kyphosis patients and control patients with chronic low back pain
Kang, C.H., Shin, M.J., Kim, S.M., Lee, S.H., Lee, C.-S.
Published in Clinical radiology (01.05.2007)
Published in Clinical radiology (01.05.2007)
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Journal Article
Imaging Flow Cytometry Analysis to Identify Differences of Survival Motor Neuron Protein Expression in Patients With Spinal Muscular Atrophy
Arakawa, Reiko, Arakawa, Masayuki, Kaneko, Kaori, Otsuki, Noriko, Aoki, Ryoko, Saito, Kayoko
Published in Pediatric neurology (01.08.2016)
Published in Pediatric neurology (01.08.2016)
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Journal Article
Evaluation of spinal and bulbar muscular atrophy by the clustering index method
Higashihara, Mana, Sonoo, Masahiro, Yamamoto, Tomotaka, Nagashima, Yu, Uesugi, Haruo, Terao, Yasuo, Ugawa, Yoshikazu, Stålberg, Erik, Tsuji, Shoji
Published in Muscle & nerve (01.10.2011)
Published in Muscle & nerve (01.10.2011)
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Journal Article
Depression in Myotonic Dystrophy type 1: clinical and neuronal correlates
Winblad, Stefan, Jensen, Christer, Månsson, Jan-Eric, Samuelsson, Lena, Lindberg, Christopher
Published in Behavioral and brain functions (19.05.2010)
Published in Behavioral and brain functions (19.05.2010)
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Journal Article
Small-fiber involvement in spinobulbar muscular atrophy (Kennedy's disease)
Manganelli, Fiore, Iodice, Valeria, Provitera, Vincenzo, Pisciotta, Chiara, Nolano, Maria, Perretti, Anna, Santoro, Lucio
Published in Muscle & nerve (01.12.2007)
Published in Muscle & nerve (01.12.2007)
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Journal Article
Gene deletions in spinal muscular atrophy
Rodrigues, N R, Owen, N, Talbot, K, Patel, S, Muntoni, F, Ignatius, J, Dubowitz, V, Davies, K E
Published in Journal of medical genetics (01.02.1996)
Published in Journal of medical genetics (01.02.1996)
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Journal Article