Discovery of Risdiplam, a Selective Survival of Motor Neuron‑2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)
Ratni, Hasane, Ebeling, Martin, Baird, John, Bendels, Stefanie, Bylund, Johan, Chen, Karen S, Denk, Nora, Feng, Zhihua, Green, Luke, Guerard, Melanie, Jablonski, Philippe, Jacobsen, Bjoern, Khwaja, Omar, Kletzl, Heidemarie, Ko, Chien-Ping, Kustermann, Stefan, Marquet, Anne, Metzger, Friedrich, Mueller, Barbara, Naryshkin, Nikolai A, Paushkin, Sergey V, Pinard, Emmanuel, Poirier, Agnès, Reutlinger, Michael, Weetall, Marla, Zeller, Andreas, Zhao, Xin, Mueller, Lutz
Published in Journal of medicinal chemistry (09.08.2018)
Published in Journal of medicinal chemistry (09.08.2018)
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Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers
Sivaramakrishnan, Manaswini, McCarthy, Kathleen D, Campagne, Sébastien, Huber, Sylwia, Meier, Sonja, Augustin, Angélique, Heckel, Tobias, Meistermann, Hélène, Hug, Melanie N, Birrer, Pascale, Moursy, Ahmed, Khawaja, Sarah, Schmucki, Roland, Berntenis, Nikos, Giroud, Nicolas, Golling, Sabrina, Tzouros, Manuel, Banfai, Balazs, Duran-Pacheco, Gonzalo, Lamerz, Jens, Hsiu Liu, Ying, Luebbers, Thomas, Ratni, Hasane, Ebeling, Martin, Cléry, Antoine, Paushkin, Sergey, Krainer, Adrian R, Allain, Frédéric H-T, Metzger, Friedrich
Published in Nature communications (14.11.2017)
Published in Nature communications (14.11.2017)
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Targeting RNA structure in SMN2 reverses spinal muscular atrophy molecular phenotypes
Garcia-Lopez, Amparo, Tessaro, Francesca, Jonker, Hendrik R A, Wacker, Anna, Richter, Christian, Comte, Arnaud, Berntenis, Nikolaos, Schmucki, Roland, Hatje, Klas, Petermann, Olivier, Chiriano, Gianpaolo, Perozzo, Remo, Sciarra, Daniel, Konieczny, Piotr, Faustino, Ignacio, Fournet, Guy, Orozco, Modesto, Artero, Ruben, Metzger, Friedrich, Ebeling, Martin, Goekjian, Peter, Joseph, Benoît, Schwalbe, Harald, Scapozza, Leonardo
Published in Nature communications (23.05.2018)
Published in Nature communications (23.05.2018)
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Journal Article
Impaired myogenic development, differentiation and function in hESC-derived SMA myoblasts and myotubes
Hellbach, Nicole, Peterson, Suzanne, Haehnke, Daniel, Shankar, Aditi, LaBarge, Samuel, Pivaroff, Cullen, Saenger, Stefanie, Thomas, Carolin, McCarthy, Kathleen, Ebeling, Martin, Hayhurst Bennett, Monica, Schmidt, Uli, Metzger, Friedrich
Published in PloS one (10.10.2018)
Published in PloS one (10.10.2018)
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Molecular, physiological, and motor performance defects in DMSXL mice carrying >1,000 CTG repeats from the human DM1 locus
Huguet, Aline, Medja, Fadia, Nicole, Annie, Vignaud, Alban, Guiraud-Dogan, Céline, Ferry, Arnaud, Decostre, Valérie, Hogrel, Jean-Yves, Metzger, Friedrich, Hoeflich, Andreas, Baraibar, Martin, Gomes-Pereira, Mário, Puymirat, Jack, Bassez, Guillaume, Furling, Denis, Munnich, Arnold, Gourdon, Geneviève
Published in PLoS genetics (01.11.2012)
Published in PLoS genetics (01.11.2012)
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Selective degeneration of septal and hippocampal GABAergic neurons in a mouse model of amyloidosis and tauopathy
Loreth, Desirée, Ozmen, Laurence, Revel, Florent G, Knoflach, Frédéric, Wetzel, Philine, Frotscher, Michael, Metzger, Friedrich, Kretz, Oliver
Published in Neurobiology of disease (01.07.2012)
Published in Neurobiology of disease (01.07.2012)
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Journal Article
PEGylated insulin-like growth factor-I affords protection and facilitates recovery of lost functions post-focal ischemia
Parker, Kim, Berretta, Antonio, Saenger, Stefanie, Sivaramakrishnan, Manaswini, Shirley, Simon A, Metzger, Friedrich, Clarkson, Andrew N
Published in Scientific reports (21.03.2017)
Published in Scientific reports (21.03.2017)
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Local application of engineered insulin-like growth factor I mRNA demonstrates regenerative therapeutic potential in vivo
Antony, Justin S., Birrer, Pascale, Bohnert, Claudia, Zimmerli, Sina, Hillmann, Petra, Schaffhauser, Hervé, Hoeflich, Christine, Hoeflich, Andreas, Khairallah, Ramzi, Satoh, Andreas T., Kappeler, Isabelle, Ferreira, Isabel, Zuideveld, Klaas P., Metzger, Friedrich
Published in Molecular therapy. Nucleic acids (12.12.2023)
Published in Molecular therapy. Nucleic acids (12.12.2023)
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Journal Article
Insulinotropic treatments exacerbate metabolic syndrome in mice lacking MeCP2 function
Pitcher, Meagan R, Ward, Christopher S, Arvide, E Melissa, Chapleau, Christopher A, Pozzo-Miller, Lucas, Hoeflich, Andreas, Sivaramakrishnan, Manaswini, Saenger, Stefanie, Metzger, Friedrich, Neul, Jeffrey L
Published in Human molecular genetics (01.07.2013)
Published in Human molecular genetics (01.07.2013)
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Journal Article
Nerve sprouting capacity in a pharmacologically induced mouse model of spinal muscular atrophy
Rimer, Mendell, Seaberg, Bonnie L, Yen, Pei-Fen, Lam, Steven, Hastings, Robert Louis, Lee, Young Il, Thompson, Wesley J, Feng, Zhihua, Metzger, Friedrich, Paushkin, Sergey, Ko, Chien-Ping
Published in Scientific reports (24.05.2019)
Published in Scientific reports (24.05.2019)
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Journal Article
Brain expression profiles of two SCN1A antisense RNAs in children and adolescents with epilepsy
Schneider, Marius Frederik, Vogt, Miriam, Scheuermann, Johanna, Müller, Veronika, Fischer-Hentrich, Antje H. L., Kremer, Thomas, Lugert, Sebastian, Metzger, Friedrich, Kudernatsch, Manfred, Kluger, Gerhard, Hartlieb, Till, Noachtar, Soheyl, Vollmar, Christian, Kunz, Mathias, Tonn, Jörg Christian, Coras, Roland, Blümcke, Ingmar, Pace, Claudia, Heinen, Florian, Klein, Christoph, Potschka, Heidrun, Borggraefe, Ingo
Published in Translational neuroscience (01.01.2024)
Published in Translational neuroscience (01.01.2024)
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Overlap of Peak Growth Activity and Peak IGF-1 to IGFBP Ratio: Delayed Increase of IGFBPs versus IGF-1 in Serum as a Mechanism to Speed up and down Postnatal Weight Gain in Mice
Walz, Michael, Chau, Luong, Walz, Christina, Sawitzky, Mandy, Ohde, Daniela, Brenmoehl, Julia, Tuchscherer, Armin, Langhammer, Martina, Metzger, Friedrich, Höflich, Christine, Hoeflich, Andreas
Published in Cells (Basel, Switzerland) (22.06.2020)
Published in Cells (Basel, Switzerland) (22.06.2020)
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Journal Article
Separation of Fast from Slow Anabolism by Site-specific PEGylation of Insulin-like Growth Factor I (IGF-I)
Metzger, Friedrich, Sajid, Waseem, Saenger, Stefanie, Staudenmaier, Christian, van der Poel, Chris, Sobottka, Bettina, Schuler, Angelika, Sawitzky, Mandy, Poirier, Raphael, Tuerck, Dietrich, Schick, Eginhard, Schaubmar, Andreas, Hesse, Friederike, Amrein, Kurt, Loetscher, Hansruedi, Lynch, Gordon S., Hoeflich, Andreas, De Meyts, Pierre, Schoenfeld, Hans-Joachim
Published in The Journal of biological chemistry (03.06.2011)
Published in The Journal of biological chemistry (03.06.2011)
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Journal Article
ProNGF inhibits NGF-mediated TrkA activation in PC12 cells
Sobottka, Bettina, Reinhardt, Dieter, Brockhaus, Manfred, Jacobsen, Helmut, Metzger, Friedrich
Published in Journal of neurochemistry (01.12.2008)
Published in Journal of neurochemistry (01.12.2008)
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Journal Article
Altered metabolic profile in the frontal cortex of PS2APP transgenic mice, monitored throughout their life span
von Kienlin, Markus, Künnecke, Basil, Metzger, Friedrich, Steiner, Guido, Richards, J. Grayson, Ozmen, Laurence, Jacobsen, Helmut, Loetscher, Hansruedi
Published in Neurobiology of disease (01.02.2005)
Published in Neurobiology of disease (01.02.2005)
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Journal Article
Phenotype selection reveals coevolution of muscle glycogen and protein and PTEN as a gate keeper for the accretion of muscle mass in adult female mice
Sawitzky, Mandy, Zeissler, Anja, Langhammer, Martina, Bielohuby, Maximilian, Stock, Peggy, Hammon, Harald M, Görs, Solvig, Metges, Cornelia C, Stoehr, Barbara J M, Bidlingmaier, Martin, Fromm-Dornieden, Carolin, Baumgartner, Bernhard G, Christ, Bruno, Brenig, Bertram, Binder, Gerhard, Metzger, Friedrich, Renne, Ulla, Hoeflich, Andreas
Published in PloS one (29.06.2012)
Published in PloS one (29.06.2012)
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Journal Article
Effect of Glutamate on Dendritic Growth in Embryonic Rat Motoneurons
Metzger, Friedrich, Wiese, Stefan, Sendtner, Michael
Published in The Journal of neuroscience (01.03.1998)
Published in The Journal of neuroscience (01.03.1998)
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