Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational study

Authors: Baum, Petra, Baumgartner, Manuela, Bertsche, Astrid, Blankenburg, Markus, Denecke, Jonas, Deschauer, Marcus, Eckenweiler, Matthias, Geis, Tobias, Goldhahn, Klaus, Groß, Martin, Günther, René, Hagenacker, Tim, Hamelmann, Eckard, Husain, Ralf A., Kamm, Christoph, Kauffmann, Birgit, Koch, Jan Christoph, Löscher, Wolfgang, Ludolph, Albert, Martin, Pascal, Mensch, Alexander, Meyer zu Hörste, Gerd, Neuwirth, Christoph, Petri, Susanne, Pühringer, Manuel, Rathmann, Imke, Schäfer, Dorothee, Schimmel, Mareike, Schrank, Bertold, Schreiber-Katz, Olivia, Schwerin-Nagel, Anette, Smitka, Martin, Steinbach, Meike, Steiner, Elisabeth, Stoffels, Johannes, Theophil, Manuela, Topakian, Raffi, Türk, Matthias, Vorgerd, Matthias, Walter, Maggie C., Weiler, Markus, Wiegand, Gert, Wunderlich, Gilbert, Wurster, Claudia Diana, Zeller, Daniel, Metelmann, Moritz, Zeiner, Fiona, Pilshofer, Veronika, Rappold, Mika, Pauschek, Josefine, Reihle, Christof, Homma, Annette Karolin, Lingor, Paul, Henzi, Bettina, Reinhardt, Tabea, Holzwarth, Dorothea, Wittmann, Wolfgang, Kappel, Stefan, Freigang, Maren, Stolte, Benjamin, Martakis, Kyriakos, Classen, Georg, Roland-Schäfer, Doris, Steuernagel, Daniela, Hartmann, Hans, Fischer, Sophie, Wermuth, Marieke, Muhandes, Mohamad Tareq, Hotter, Anna, Uzelac, Zeljko, Naegel, Steffen, Wiethoff, Sarah, Braun, Nathalie, Bjelica, Bogdan, Kölbel, Heike, Angelova-Toshkina, Daniela, Wilken, Bernd, Osmanovic, Alma, Fiedler, Barbara, Plecko, Barbara, Tomforde, Maike, Voelkl, Thomas, von Moers, Arpad, Müller, Petra, Behring, Bettina, Güttsches, Anne, Reilich, Peter, Wick, Wolfgang, Stoltenburg, Corinna, Witzel, Simon, Bellut, Julia, Hoffmann, Georg Friedrich, Mörtlbauer, Kathrin, Ille, Alexandra, Schroth, Michael, Driemeyer, Joenna, Semmler, Luisa, Müller, Cornelia, Dörnbrack, Katharina, Zemlin, Michael, Geitmann, Stephanie, Lapp, Hanna Sophie, Brakemeier, Svenja, Gehrke, Tascha, Ntemiris, Klearchos, Kaiser, Nadja, Borowski, Sabine, Ramadan, Barbara, Hustedt, Ulf, Baum, Tobias, Schneider, Ilka, Akova-Oztürk, Esra, Vill, Katharina, Dibrani, Zylfie, Wohnrade, Camilla, Della-Marina, Adela, Jung, Lisa, Deba, Timo, Zobel, Joachim, Schallner, Jens, Kraut, Christina, Vollmann, Peter, Schüssler, Stephanie, Roeder, Melanie, Hiebeler, Miriam, Berberich, Nicole, Schneider, Joanna, Brauner, Brigitte, Kölker, Stefan, Pernegger, Elke, Gosk-Tomek, Magdalena, Braun, Sarah, Weiss, Deike, Machetanz, Gerrit, Langer, Thorsten, Saier, Christina, Baumann, Sandra, Hettrich, Sabine, Dworschak, Gabriel, Müller-Kaempffer, Katharina, Dittes, Isabelle, Thimm, Andreas, Quinten, Lisa, Albers, Kristina, Bevot, Andrea, Bretschneider, Christa, Dorst, Johannes, Kendzierski, Thomas, Hannibal, Iris, Bischofberger, Jasmin, Riesmeier, Tilman, Gangfuß, Andrea, Johann to Settel, Eva, Grässl, Michael, Fiebig, Susan, Hollerauer, Carmen, Seeber, Lea, Krahwinkler, Ina, Lange, Irene, Montagnese, Federica, Mann-Richter, Marcel, Wagner, Alexandra, Leypold, Christine, Saffari, Afshin, Anna, Elmecker, Wiesenhofer, Anna, Wendel, Eva-Maria, Steffens, Paula-Sophie, Wider, Sabine, Tassoni, Adrian, Dall, Andrea, Busch, Franziska, Zeisler, Daniela, Wessel, Maria, Lipka, Jaqueline, Hackemer, Andrea, Plugge, Loreen, Jansen, Eva, Roth, Erdmute, Schuster, Joachim, Koelsch, Anna, Warken-Madelung, Birgit, Schwippert, Michaela, Holtkamp, Britta, Köbbing, Katja, Claeys, Sander, Foerster, Sandy, Thiele, Simone, Rochau-Trumpp, Heidi, George, Annette, Niesert, Moritz, Neimair, Tanja, Vettori, Katia, Haverkamp, Julia, Taherpour, Jila, Hug, Juliane, Wenzel, Franziska, Bant, Christina, Baur, Ute, Bühner, Kathrin, Schlag, Melina, Ruß, Lena, Küpper, Hanna, Müller, Anja, Wollinsky, Kurt, Well, Therese, Leinert, Antonia, Andres, Barbara, Omran, Heymut, Claus, Nicole, Hagenmeyer, Anna, Schnurr, Marion, Dukic, Vladimir, Ludolph, Albert Christian, Specht, Sabine, Angermair, Verena, Hüpper, Anna, Banholzer, Daniela, Stein, Sabine, Kampowski, Tim, Richmann, Marion, Nicolai, Sylke, Atta, Omar, Meßmer, Birgit, de Vries, Heike, Rotenfusser, Elisabeth, Oscmanovic, Alma, Renger, Isabelle, Guillemot, Hélène, Lehnert, Ilka, Grünwedel, Mike, Grimm, Laura, Stocker, Guido, Kaindl, Angela M., Hoevel, Annegret, Stadler, Theresa, Fischer, Michal, Vogt, Sibylle, Gebert, Axel, Goldbach, Susanne, Kirschner, Janbernd, Lochmüller, Hanns, Müller-Felber, Wolfgang, Pechmann, Astrid, Schara-Schmidt, Ulrike, Probst-Schendzielorz, Kristina, Lang, Annina, Nitzsche, Maren, Hammer, Julie, Müller-Kaempfer, Katharina, Wirner-Piotrowski, Corinna, van der Stam, Lieske, Bongartz, Anke, Enzmann, Cornelia, Fluss, Joël, Galiart, Elea, Jacquier, David, Metzler, Dominique Baumann, Tscherter, Anne, Weiß, Claudia, Becker, Lena-Luise, Friese, Johannes, Blaschek, Astrid, Hahn, Andreas, Illsinger, Sabine, Schwartz, Oliver, Bernert, Günther, Hagen, Maja von der, Flotats-Bastardas, Marina, Schreiber, Gudrun, Schara, Ulrike, Trollmann, Regina, Horber, Veronka, Wilichowski, Ekkehard, Baumann, Matthias, Klein, Andrea, Eisenkölbl, Astrid, Köhler, Cornelia, Stettner, Georg M., Cirak, Sebahattin, Hasselmann, Oswald, Garbade, Sven F., Johannsen, Jessika, Ziegler, Andreas

Source: In The Lancet Regional Health - Europe December 2024 47

Antikörper-basierte Immuntherapien gegen Morbus Parkinson

Authors: Machetanz, Gerrit, Wolff, Andreas, Lingor, Paul

Source: Psychopharmakotherapie. 29(2):56-63

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN628850367&indx=1&recIds=ETOCRN628850367

The longevity gene Klotho and its cerebrospinal fluid protein profiles as a modifier for Parkinson´s disease

Authors: Zimmermann, Milan, Köhler, Leonie, Kovarova, Marketa, Lerche, Stefanie, Schulte, Claudia, Wurster, Isabel, Machetanz, Gerrit, Deuschle, Christian, Hauser, Ann‐Kathrin, Gasser, Thomas, Berg, Daniela, Schleicher, Erwin, Maetzler, Walter, Brockmann, Kathrin

Source: European journal of neurology. 28(5):1557-1565

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100127419889.0x000001&indx=1&recIds=ETOCvdc_100127419889.0x000001

The Mutation Matters: CSF Profiles of GCase, Sphingolipids, α‐Synuclein in PDGBA

Authors: Lerche, Stefanie, Schulte, Claudia, Wurster, Isabel, Machetanz, Gerrit, Roeben, Benjamin, Zimmermann, Milan, Deuschle, Christian, Hauser, Ann‐Kathrin, Böhringer, Judith, Krägeloh‐Mann, Ingeborg, Waniek, Katharina, Lachmann, Ingolf, Petterson, Xuan‐Mai T., Chiang, Ruby, Park, Hyejung, Wang, Bing, Liepelt‐Scarfone, Inga, Maetzler, Walter, Galasko, Douglas, Scherzer, Clemens R., Gasser, Thomas, Mielke, Michelle M., Hutten, Samantha J., Mollenhauer, Brit, Sardi, S. Pablo, Berg, Daniela, Brockmann, Kathrin

Source: Movement disorders. 36(5):1216-1228

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100164604272.0x000001&indx=1&recIds=ETOCvdc_100164604272.0x000001

The Progressive Supranuclear Palsy Clinical Deficits Scale

Authors: Piot, Ines, Schweyer, Kerstin, Respondek, Gesine, Stamelou, Maria, Sckopke, Philipp, Schenk, Thomas, Goetz, Christopher G., Stebbins, Glenn T., Höglinger, Günter U., Gasser, Thomas, Hermann, Andreas, Höglinger, Günter, Höllerhage, Matthias, Kimmich, Okka, Klockgether, Thomas, Levin, Johannes, Machetanz, Gerrit, Osterrath, Antje, Palleis, Carla, Prudlo, Johannes, Spottke, Annika, Berg, Daniela, Bürk, Katrin, Claßen, Joseph, Eggers, Carsten, Greuel, Andrea, Grimm, Max‐Joseph, Hermann, Lennard, Iankova, Vassilena, Jahn, Klaus, Jost, Wolfgang, Klietz, Martin, Kühn, Andrea, Marxreiter, Franz, Paschen, Steffen, Poetter‐Nerger, Monika, Preisl, Marie‐Therese, Prilop, Lisa, Tönges, Lars, Trenkwalder, Claudia, Warnecke, Tobias, Wegner, Florian, Winkler, Jürgen, Antonini, Angelo, P, Kailash P., L, Adam L., Colosimo, Carlo, Compta, Yaroslau, Corvol, Jean‐Christophe, I, Lawrence I., E, Anthony E., Litvan, Irene, R, Huw R., Nilsson, Christer, Pantelyat, Alexander

Source: Movement disorders. 35(4):650-661

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100131552807.0x000001&indx=1&recIds=ETOCvdc_100131552807.0x000001

CSF NFL in a Longitudinally Assessed PD Cohort: Age Effects and Cognitive Trajectories

Authors: Lerche, Stefanie, Wurster, Isabel, Röben, Benjamin, Zimmermann, Milan, Machetanz, Gerrit, Wiethoff, Sarah, Dehnert, Monique, Rietschel, Lea, Riebenbauer, Benjamin, Deuschle, Christian, Stransky, Elke, Lieplt‐Scarfone, Inga, Gasser, Thomas, Brockmann, Kathrin

Source: Movement disorders. 35(7):1138-1144

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100107588462.0x000001&indx=1&recIds=ETOCvdc_100107588462.0x000001

Uncovering genetic mimics in multiple sclerosis: A single-center clinical exome sequencing study.

Authors: Mandler, Julia M., Härtl, Johanna, Cordts, Isabell, Sturm, Marc, Hedderich, Dennis M., Bafligil, Cemsel, Baki, Enayatullah, Becker, Benedikt, Machetanz, Gerrit, Haack, Tobias B., Berthele, Achim, Hemmer, Bernhard, Deschauer, Marcus

Source: Multiple Sclerosis Journal - Experimental, Translational & Clinical; 7/24/2024, p1-12, 12p

Subject Terms: MULTIPLE sclerosis, GENETIC testing, FAMILY history (Medicine), DIFFERENTIAL diagnosis, LEUKOENCEPHALOPATHIES

Patients' and caregivers' perception of multidimensional and palliative care in amyotrophic lateral sclerosis – protocol of a German multicentre study.

Authors: Linse, Katharina, Weber, Constanze, Reilich, Peter, Schöberl, Florian, Boentert, Matthias, Petri, Susanne, Rödiger, Annekathrin, Posa, Andreas, Otto, Markus, Wolf, Joachim, Zeller, Daniel, Brunkhorst, Robert, Koch, Jan, Hermann, Andreas, Großkreutz, Julian, Schröter, Carsten, Groß, Martin, Lingor, Paul, Machetanz, Gerrit, Semmler, Luisa

Source: Neurological Research & Practice; 7/4/2024, Vol. 6 Issue 1, p1-8, 8p

Subject Terms: CAREGIVER attitudes, MOTOR neuron diseases, PALLIATIVE treatment, BURDEN of care, PATIENTS, AMYOTROPHIC lateral sclerosis, MEDICAL care, SPIRITUAL healing

Geographic Terms: GERMANY

Deterioration of executive dysfunction in elderly with REM sleep behavior disorder (RBD)

Authors: Lerche, Stefanie, Machetanz, Gerrit, Roeben, Benjamin, Wurster, Isabel, Zimmermann, Milan, von Thaler, Anna-Katharina, Liepelt-Scarfone, Inga, Eschweiler, Gerhard W., Fallgatter, Andreas, Metzger, Florian, Maetzler, Walter, Berg, Daniela, Brockmann, Kathrin

Source: In Neurobiology of Aging October 2018 70:242-246

Dementia with lewy bodies: GBA1 mutations are associated with cerebrospinal fluid alpha‐synuclein profile

Authors: Lerche, Stefanie, Machetanz, Gerrit, Wurster, Isabel, Roeben, Benjamin, Zimmermann, Milan, Pilotto, Andrea, Preische, Oliver, Stransky, Elke, Deuschle, Christian, Hauser, Ann‐Kathrin, Schulte, Claudia, Lachmann, Ingolf, Waniek, Katharina, Gasser, Thomas, Berg, Daniela, Maetzler, Walter, Brockmann, Kathrin

Source: Movement disorders. 34(7):1069-1073

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100095112875.0x000001&indx=1&recIds=ETOCvdc_100095112875.0x000001

Validation of a novel Montreal Cognitive Assessment scoring algorithm in non-demented Parkinson's disease patients

Authors: Sulzer, Patricia, Becker, Sara, Maetzler, Walter, Kalbe, Elke, van Nueten, Luc, Timmers, Maarten, Machetanz, Gerrit, Streffer, Johannes, Salvadore, Giacomo, Scholz, Erich, Tkaczynska, Zuzanna, Brockmann, Kathrin, Berg, Daniela, Liepelt-Scarfone, Inga

Source: Journal of neurology. 265(9):1976-1984

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100141523225.0x000001&indx=1&recIds=ETOCvdc_100141523225.0x000001

Symptomatic intracranial hypertension in an adult patient with spinal muscular atrophy and arachnoid cysts receiving nusinersen.

Authors: Machetanz, Gerrit, Grziwotz, Marc, Semmler, Luisa, Maier, Mathias, Maegerlein, Christian, Deschauer, Marcus

Source: Journal of Neuromuscular Diseases; 2023, Vol. 10 Issue 4, p719-725, 7p

Parkinson's disease therapy: what lies ahead?

Authors: Wolff, Andreas, Schumacher, Nicolas U., Pürner, Dominik, Machetanz, Gerrit, Demleitner, Antonia F., Feneberg, Emily, Hagemeier, Maike, Lingor, Paul

Source: Journal of Neural Transmission; Jun2023, Vol. 130 Issue 6, p793-820, 28p

Subject Terms: PARKINSON'S disease, MOVEMENT disorders, DRUG development, LIFE expectancy, DISEASE duration, DISEASE progression

Current State and Future Directions in the Diagnosis of Amyotrophic Lateral Sclerosis.

Authors: Vidovic, Maximilian, Müschen, Lars Hendrik, Brakemeier, Svenja, Machetanz, Gerrit, Naumann, Marcel, Castro-Gomez, Sergio

Source: Cells (2073-4409); Mar2023, Vol. 12 Issue 5, p736, 24p

Subject Terms: AMYOTROPHIC lateral sclerosis, DIAGNOSIS, THERAPEUTICS, SKELETAL muscle, DELAYED diagnosis, MOTOR neuron diseases

The Mutation Matters: CSF Profiles of GCase, Sphingolipids, α‐Synuclein in PDGBA.

Authors: Lerche, Stefanie, Schulte, Claudia, Wurster, Isabel, Machetanz, Gerrit, Roeben, Benjamin, Zimmermann, Milan, Deuschle, Christian, Hauser, Ann‐Kathrin, Böhringer, Judith, Krägeloh‐Mann, Ingeborg, Waniek, Katharina, Lachmann, Ingolf, Petterson, Xuan‐Mai T., Chiang, Ruby, Park, Hyejung, Wang, Bing, Liepelt‐Scarfone, Inga, Maetzler, Walter, Galasko, Douglas, Scherzer, Clemens R.

Source: Movement Disorders; May2021, Vol. 36 Issue 5, p1216-1228, 13p

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Parkinson's disease: evolution of cognitive impairment and CSF Aβ1-42 profiles in a prospective longitudinal study.

Authors: Lerche, Stefanie, Wurster, Isabel, Röben, Benjamin, Machetanz, Gerrit, Zimmermann, Milan, Bernhard, Felix, Stransky, Elke, Deuschle, Christian, Schulte, Claudia, Hansson, Oskar, Zetterberg, Henrik, Gasser, Thomas, Berg, Daniela, Maetzler, Walter, Brockmann, Kathrin

Source: Journal of Neurology, Neurosurgery & Psychiatry; Feb2019, Vol. 90 Issue 2, p165-170, 6p, 5 Charts, 2 Graphs

Subject Terms: CEREBROSPINAL fluid, UNILATERAL neglect, PARKINSON'S disease, LONGITUDINAL method

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Parkinson’s disease: evolution of cognitive impairment and CSF Aβ1–42profiles in a prospective longitudinal study

Authors: Lerche, Stefanie, Wurster, Isabel, Ro¨ben, Benjamin, Machetanz, Gerrit, Zimmermann, Milan, Bernhard, Felix, Stransky, Elke, Deuschle, Christian, Schulte, Claudia, Hansson, Oskar, Zetterberg, Henrik, Gasser, Thomas, Berg, Daniela, Maetzler, Walter, Brockmann, Kathrin

Source: Journal of Neurology, Neurosurgery, & Psychiatry (JNNP); 2019, Vol. 90 Issue: 2 p165-170, 6p