Long-term efficacy and safety of nusinersen in adults with 5q spinal muscular atrophy: a prospective European multinational observational study

Autor: Günther, René, Wurster, Claudia Diana, Brakemeier, Svenja, Osmanovic, Alma, Schreiber-Katz, Olivia, Petri, Susanne, Uzelac, Zeljko, Hiebeler, Miriam, Thiele, Simone, Walter, Maggie C., Weiler, Markus, Kessler, Tobias, Freigang, Maren, Lapp, Hanna Sophie, Cordts, Isabell, Lingor, Paul, Deschauer, Marcus, Hahn, Andreas, Martakis, Kyriakos, Steinbach, Robert, Ilse, Benjamin, Rödiger, Annekathrin, Bellut, Julia, Nentwich, Julia, Zeller, Daniel, Muhandes, Mohamad Tareq, Baum, Tobias, Christoph Koch, Jan, Schrank, Bertold, Fischer, Sophie, Hermann, Andreas, Kamm, Christoph, Naegel, Steffen, Mensch, Alexander, Weber, Markus, Neuwirth, Christoph, Lehmann, Helmar C., Wunderlich, Gilbert, Stadler, Christian, Tomforde, Maike, George, Annette, Groß, Martin, Pechmann, Astrid, Kirschner, Janbernd, Türk, Matthias, Schimmel, Mareike, Bernert, Günther, Martin, Pascal, Rauscher, Christian, Meyer zu Hörste, Gerd, Baum, Petra, Löscher, Wolfgang, Flotats-Bastardas, Marina, Köhler, Cornelia, Probst-Schendzielorz, Kristina, Goldbach, Susanne, Schara-Schmidt, Ulrike, Müller-Felber, Wolfgang, Lochmüller, Hanns, von Velsen, Otgonzul, Kleinschnitz, Christoph, Ludolph, Albert C., Hagenacker, Tim

Publikováno v: In The Lancet Regional Health - Europe April 2024 39

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Increased chitotriosidase 1 concentration following nusinersen treatment in spinal muscular atrophy

Autor: Freigang, Maren, Steinacker, Petra, Otto, Markus, Hermann, Andreas, Günther, René, Wurster, Claudia Diana, Schreiber-Katz, Olivia, Osmanovic, Alma, Petri, Susanne, Koch, Jan Christoph, Rostásy, Kevin, Falkenburger, Björn, Ludolph, Albert

Publikováno v: Orphanet Journal of Rare Diseases, Vol 16, Iss 1, Pp 1-11 (2021)
Orphanet Journal of Rare Diseases
Orphanet journal of rare diseases 16(1), 330 (2021). doi:10.1186/s13023-021-01961-8

Background Studies regarding the impact of (neuro)inflammation and inflammatory response following repetitive, intrathecally administered antisense oligonucleotides (ASO) in 5q-associated spinal muscular atrophy (SMA) are sparse. Increased risk of hy

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid_dedup__::c1f62fdc8a2e14a874cbf8a1adc782fb
https://doaj.org/article/0cde2deffee34edd83dfefc691547d42

Additional file 1 of Increased chitotriosidase 1 concentration following nusinersen treatment in spinal muscular atrophy

Autor: Freigang, Maren, Steinacker, Petra, Wurster, Claudia Diana, Schreiber-Katz, Olivia, Osmanovic, Alma, Petri, Susanne, Koch, Jan Christoph, Rostásy, Kevin, Falkenburger, Björn, Ludolph, Albert Christian, Otto, Markus, Hermann, Andreas, Günther, René

Additional file 1: Table S1. Subgroup characteristics. IQR interquartile range.

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c199f98d6a281884f3db4ec3921ab2da

Patient-Reported Prevalence of Non-motor Symptoms Is Low in Adult Patients Suffering From 5q Spinal Muscular Atrophy.

Autor: Günther, René, Wurster, Claudia Diana, Cordts, Isabell, Koch, Jan Christoph, Kamm, Christoph, Petzold, Daniel, Aust, Elisa, Deschauer, Marcus, Lingor, Paul, Ludolph, Albert Christian, Hermann, Andreas

Publikováno v: Frontiers in Neurology; 11/1/2019, Vol. 10, p1-7, 7p