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  1. Ludwanowski, Simon [Author]; Ari, Meral [Author]; Parison, Karsten [Author]; Kalthoum, Somar [Author]; Straub, Paula S. [Author]; Pompe, Nils [Author]; Weber, Stefan [Author]; Walter, Michael [Author]; Walther, Andreas [Author]

    pH Tuning of water‐soluble Arylazopyrazole photoswitches

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    Weinheim: Wiley, 2020

    Published in: Chemistry – a European journal ; 26, 58 (2020), 13203-13212

  2. Anthony, Karen; Cirak, Sebahattin; Torelli, Silvia; Tasca, Giorgio; Feng, Lucy; Arechavala-Gomeza, Virginia; Armaroli, Annarita; Guglieri, Michela; Straathof, Chiara S.; Verschuuren, Jan J.; Aartsma-Rus, Annemieke; Helderman-van den Enden, Paula; Bushby, Katherine; Straub, Volker; Sewry, Caroline; Ferlini, Alessandra; Ricci, Enzo; Morgan, Jennifer E.; Muntoni, Francesco

    Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials

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    Oxford University Press (OUP), 2011

    Published in: Brain, 134 (2011) 12, Seite 3547-3559

  3. Dang, Utkarsh J.; Damsker, Jesse M.; Guglieri, Michela; Clemens, Paula R.; Perlman, Seth J.; Smith, Edward C.; Horrocks, Iain; Finkel, Richard S.; Mah, Jean K.; Deconinck, Nicolas; Goemans, Nathalie M.; Haberlová, Jana; Straub, Volker; Mengle-Gaw, Laurel; Schwartz, Benjamin D.; Harper, Amy; Shieh, Perry B.; De Waele, Liesbeth; Castro, Diana; Yang, Michele L.; Ryan, Monique M.; McDonald, Craig M.; Tulinius, Mar; Webster, Richard I.; [...]

    Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy : A Randomized Controlled Trial : A Randomized Controlled Trial

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    Ovid Technologies (Wolters Kluwer Health), 2024

    Published in: Neurology, 102 (2024) 5

  4. Guglieri, Michela; Clemens, Paula R.; Perlman, Seth J.; Smith, Edward C.; Horrocks, Iain; Finkel, Richard S.; Mah, Jean K.; Deconinck, Nicolas; Goemans, Nathalie; Haberlova, Jana; Straub, Volker; Mengle-Gaw, Laurel J.; Schwartz, Benjamin D.; Harper, Amy D.; Shieh, Perry B.; De Waele, Liesbeth; Castro, Diana; Yang, Michelle L.; Ryan, Monique M.; McDonald, Craig M.; Tulinius, Mar; Webster, Richard; McMillan, Hugh J.; Kuntz, Nancy L.; [...]

    Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy : A Randomized Clinical Trial : A Randomized Clinical Trial

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    American Medical Association (AMA), 2022

    Published in: JAMA Neurology, 79 (2022) 10, Seite 1005

  5. Bello, Luca; Flanigan, Kevin M.; Weiss, Robert B.; Spitali, Pietro; Aartsma-Rus, Annemieke; Muntoni, Francesco; Zaharieva, Irina; Ferlini, Alessandra; Mercuri, Eugenio; Tuffery-Giraud, Sylvie; Claustres, Mireille; Straub, Volker; Lochmüller, Hanns; Barp, Andrea; Vianello, Sara; Pegoraro, Elena; Punetha, Jaya; Gordish-Dressman, Heather; Giri, Mamta; McDonald, Craig M.; Hoffman, Eric P.; Dunn, Diane M.; Swoboda, Kathryn J.; Gappmaier, Eduard; [...]

    Association Study of Exon Variants in the NF-κB and TGFβ Pathways Identifies CD40 as a Modifier of Duchenne Muscular Dystrophy

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    Elsevier BV, 2016

    Published in: The American Journal of Human Genetics, 99 (2016) 5, Seite 1163-1171

  6. Mah, Jean K.; Clemens, Paula R.; Guglieri, Michela; Smith, Edward C.; Finkel, Richard S.; Tulinius, Mar; Nevo, Yoram; Ryan, Monique M.; Webster, Richard; Castro, Diana; Kuntz, Nancy L.; McDonald, Craig M.; Damsker, Jesse M.; Schwartz, Benjamin D.; Mengle-Gaw, Laurel J.; Jackowski, Stefan; Stimpson, Georgia; Ridout, Deborah A.; Ayyar-Gupta, Vandana; Baranello, Giovanni; Manzur, Adnan Y.; Muntoni, Francesco; Gordish-Dressman, Heather; Leinonen, Mika; [...]

    Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy : A 30-Month Nonrandomized Controlled Open-Label Extension Trial : A 30-Month Nonrandomized Controlled Open-Label Extension Trial

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    American Medical Association (AMA), 2022

    Published in: JAMA Network Open, 5 (2022) 1, Seite e2144178

  7. Kishnani, Priya S.; Diaz-Manera, Jordi; Toscano, Antonio; Clemens, Paula R.; Ladha, Shafeeq; Berger, Kenneth I.; Kushlaf, Hani; Straub, Volker; Carvalho, Gerson; Mozaffar, Tahseen; Roberts, Mark; Attarian, Shahram; Chien, Yin-Hsiu; Choi, Young-Chul; Day, John W.; Erdem-Ozdamar, Sevim; Illarioshkin, Sergey; Goker-Alpan, Ozlem; Kostera-Pruszczyk, Anna; van der Ploeg, Ans T.; An Haack, Kristina; Huynh-Ba, Olivier; Tammireddy, Swathi; Thibault, Nathan; [...]

    Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks : A Phase 3 Randomized Clinical Trial : A Phase 3 Randomized Clinical Trial

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    American Medical Association (AMA), 2023

    Published in: JAMA Neurology, 80 (2023) 6, Seite 558

  8. Wildenauer, Dieter B.; Schwab, Sibylle G.; Albus, Margot; Hallmayer, Joachim; Lerer, Bernard; Maier, Wolfgang; Blackwood, Douglas; Muir, Walter; St Clair, David; Morris, Stewart; Moises, Hans W.; Yang, Liu; Kristbjarnarson, Helgi; Helgason, Tomas; Wiese, Claudia; Collier, David A.; Holmans, Peter; Daniels, Jo; Rees, Mark; Asherson, Philip; Roberts, Queta; Cardno, Alastair; Arranz, Maria J.; Vallada, Homero; [...]

    Additional support for schizophrenia linkage on chromosomes 6 and 8: A multicenter study

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    Wiley, 1996

    Published in: American Journal of Medical Genetics, 67 (1996) 6, Seite 580-594

  9. Bharucha, A.; Bigi, I. I.; Bobeth, C.; Bobrowski, M.; Brod, J.; Buras, A. J.; Davies, C. T. H.; Datta, A.; Delaunay, C.; Descotes-Genon, S.; Ellis, J.; Feldmann, T.; Fleischer, R.; Gedalia, O.; Girrbach, J.; Guadagnoli, D.; Hiller, G.; Hochberg, Y.; Hurth, T.; Isidori, G.; Jäger, S.; Jung, M.; Kagan, A.; Kamenik, J. F.; [...]

    Implications of LHCb measurements and future prospects

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    Springer Science and Business Media LLC, 2013

    Published in: The European Physical Journal C, 73 (2013) 4

  10. Depond, Charles Champeaux; Zouaoui, Sonia; Darlix, Amélie; Rigau, Valérie; Mathieu-Daudé, Hélène; Bauchet, Fabienne; Khettab, Mohamed; Trétarre, Brigitte; Figarella-Branger, Dominique; Taillandier, Luc; Boetto, Julien; Pallud, Johan; Zemmoura, Ilyess; Roche, Pierre-Hugues; Bauchet, Luc; Lahoud, G. Abi; Aboukais, R.; Achim, V.; Adetchessi, T.; Aesch, B.; Agha, M.; Aghakhani, N.; Al Hallak, R.; Albert, A.; [...]

    Descriptive epidemiology of 30,223 histopathologically confirmed meningiomas in France: 2006–2015

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    Springer Science and Business Media LLC, 2024

    Published in: Acta Neurochirurgica, 166 (2024) 1

  11. Kurella Tamura, Manjula; Pajewski, Nicholas M.; Bryan, R. Nick; Weiner, Daniel E.; Diamond, Matthew; Van Buren, Peter; Taylor, Addison; Beddhu, Srinivasan; Rosendorff, Clive; Jahanian, Hesamoddin; Zaharchuk, Greg; Whelton, Paul; Johnson, Karen C.; Fine, Lawrence; Snyder, Joni; Bild, Diane; Bonds, Denise; Cook, Nakela; Cutler, Jeffrey; Czajkowski, Susan; Fine, Lawrence; Kaufmann, Peter; Kimmel, Paul; Launer, Lenore; [...]

    Chronic kidney disease, cerebral blood flow, and white matter volume in hypertensive adults

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    Ovid Technologies (Wolters Kluwer Health), 2016

    Published in: Neurology, 86 (2016) 13, Seite 1208-1216

  12. Gorman, Kathleen M.; Meyer, Esther; Grozeva, Detelina; Spinelli, Egidio; McTague, Amy; Sanchis-Juan, Alba; Carss, Keren J.; Bryant, Emily; Reich, Adi; Schneider, Amy L.; Pressler, Ronit M.; Simpson, Michael A.; Debelle, Geoff D.; Wassmer, Evangeline; Morton, Jenny; Sieciechowicz, Diana; Jan-Kamsteeg, Eric; Paciorkowski, Alex R.; King, Mary D.; Cross, J. Helen; Poduri, Annapurna; Mefford, Heather C.; Scheffer, Ingrid E.; Haack, Tobias B.; [...]

    Bi-allelic Loss-of-Function CACNA1B Mutations in Progressive Epilepsy-Dyskinesia

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    Elsevier BV, 2019

    Published in: The American Journal of Human Genetics, 104 (2019) 5, Seite 948-956