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Janbernd Kirschner

university of freiburg

64H指数
412论文数
2.1W被引数
收录论文 93
发表时间
Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland – part II – expert based recommendations for surveillance and management of side-effects德国、奥地利和瑞士关于使用onasemnogene abeparvovec进行脊髓性肌萎缩基因治疗的德尔菲共识——第二部分——基于专家的建议:副作用监测和管理
err2026-08-27
err0
PREAI
errAndreas Ziegler; Claudia Weiß; Katharina Vill; Matthias Baumann; Günther Bernert; Astrid Blaschek; Astrid Eisenkölbl; Marina Flotats-Bastardas; Johannes Friese; Dorothea Holzwarth; Claudia Ganter; Klaus Goldhahn; Andreas Hahn; Maja von der Hagen; Hans Hartmann; Oswald Hasselmann; Veronka Horber; Ralf A Husain; Sabine Illsinger; David Jacquier; Jessika Johannsen; Cornelia Köhler; Heike Kölbel; Michael Kolodzig; Andrea Klein; Astrid Pechmann; Arpad von Moers; Wolfgang Müller-Felber; Christian Rauscher; Gudrun Schreiber; Oliver Schwartz; Joachim Sproß; Georg M Stettner; Corinna Stoltenburg; Eva Stumpe; Regina Trollmann; Gerd Wiegand; Ekkehard Wilichowski; Ulrike Schara; Janbernd Kirschner; Carsten Müller-Tidow; Georg Friedrich Hoffmann; Peter Dreger; Maria-Luisa Schubert; Andreas Ziegler; Stefan Kölker; Thomas Opladen; Ulrike Schara-Schmidt; Nina Rademacher; Bastian von Tresckow; Christian Reinhardt; Marcel Teichert; Veronka Horber; Hendrik Rosewich; Samuel Gröschel; Wolfgang Bethge; Claudia Lengerke; Bärbel Entrich; Claudia Weiß; Angela M. Kaindl; Joanna Schneider; Lisa-Valerie Bitzan; Antonia Busse; Lars Bullinger; Olaf Penack; Maja von der Hagen; Martin Smitka; Josephine Schädlich; Malte von Bonin; Martin Bornhäuser; Raphael Teipel; Astrid Blaschek; Katharina Vill; Wolfgang Müller-Felber; Moritz Tacke; Fabian Hauck; Christoph Klein; Veit Bücklein; Marion Subklewe; Michael von Bergwelt; Regina Trollmann; Fabian Müller; Andreas Mackensen; Dennis Bene; Jessika Johannsen; Ania C. Muntau; Jonas Denecke; Francis Ayuketang Ayuk; Silke Heidenreich; Gesine Bug; Hubert Serve; Julia Riemann; Eva Wagner-Drouet; Beate Hauptrock; Oliver Schwartz; Hélène Guillemot; Heymut Omran; Simon Call; Matthias Stelljes; Georg Lenz; Anna Ossami Saidy; Bertram Glaß; Aleksandar Radujkovic; Sascha Dietrich; Ben-Niklas Bärmann; Friedrich Stölzel; Claudia Baldus; Natalie Schub; Sabine Illsinger; Dieter Haffner; Hans Hartmann; Astrid Pechmann; Janbernd Kirschner; Lutz Hein; Dorothea Holzwarth; Alexandra Klotz; Franziska Busch; Lisa Allenstein; Gabriel Dworschak; Andreas A. Hahn; Bernd A. Neubauer; Marina Flotats-Bastardas; Michael Zemlin; Klaus Goldhahn; Arpad von Moers; Ellen Knierim; Christian Friese; Gudrun Schreiber; Bernd Wilken; Cornelia Köhler; Thomas Lücke; Christoph Hanefeld; Sebahattin Cirak; Antje Westhoff; Kübra Erden; Lisa Benedikter; Sandra Zinke; Gerald Wulf; Wolfram Jung; Justin Hasenkamp
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Treatment evolution in spinal muscular atrophy: insights from the SMArtCARE registry
errBrain
IF11.7
err2025-12-23
err0
errOAAI
errCornelia Voigt-Müller; Michelle Pfaffenlehner; Günther Bernert; Hakan Cetin; Tim Hagenacker; Heike Kölbel; Hanns Lochmüller; Christian Pfeuffer; Katharina Vill; Maggie C Walter; Janbernd Kirschner; Astrid Pechmann; ; Pascal Martin; Christoph Neuwirth; Andreas Merkenschlager; René Günther; Mathias Müller; Regina Trollmann; Gert Wiegand; Sabine Illsinger; Omar Keritam; Matthias Baumann; Sarah Bernsen; Claudia Weiß; Gerd Meyer zu Hörste; Matthias Türk; Daniel Zeller; Thomas M K Völkl; Uta Diebold; Verena Haug; Wolfgang Löscher; Dorothea Holzwarth; Mareike Schimmel; Burkhard Stüve; Karin Wabnegger; Ralf A Husain; Oliver Schwartz; Matthias Vorgerd; Eva Matzker; Gilbert Wunderlich; Raffi Topakian; Eckard Hamelmann; Anette Schwerin-Nagel; Marina Flotats-Bastardas; Astrid Blaschek; Christof Reihle; Astrid Bertsche; Ruth Janßen; Andreas Merkenschlager; Sandra Nebgen; Martin Smitka; Andreas Ziegler; Manuela Theophil; Ruth Janßen; Moritz Metelmann; Birgit Kauffmann; Andreas Hahn; Gudrun Schreiber; Anette Schwerin-Nagel; Astrid Eisenkölbl; Martin Winterholler; Eva Stögmann; Veronka Horber; Friedrich Ebinger; Tobias Geis; Martin Fleger; Jessika Johannsen; Meike Bettina Göricke; Veronika Pilshofer; Maren Fuchs; Paul Lingor; Alexander Mensch; Robert Steinbach; Sebastian Friedrich; Julian Großkreutz; Zeljko Uzelac; Friederike Sophie Kohlmann; Harald Faninger; Simone Mahal; Christoph Korenke; Harald Binder; Katharina Dörnbrack; Jasmin Bischofberger; Janina Gburek-Augustat; Hanna Sophie Lapp; Tanja Zindler; Bettina Behring; Miriam Hiebeler; Hans Hartmann; Benjamin Stolte; Bernhard Fasching; Christian Lechner; Patrick Weydt; Joanna Schneider; Sarah Wiethoff; Julia Bellut; Johannes C Stoffels; Antje Schmidt; Corrine G C Horlings; Alexandra Klotz; Daniela Angelova-Toshkina; Sabine Knafl-Slamanig; Daniela Steuernagel; Hélène Guillemot; Melanie Drabant; Kathrin Hornung; Hormos Salimi Dafsari; Petra Müller; Georg Classen; Michael Grässl; Viola Horneff; Sarah Braun; Josefine Lendeckel; Oliver Summ; Janina Gburek-Augustat; Ilona Krauspe-Stübecke; Maja von der Hagen; Michal Fischer; Klaus Goldhahn; Oliver Summ; Christa-Caroline Bergner; Adela Della Marina; Marieke Ziegler; Omar Keritam; Kyriakos Martakis; Bernd Wilken; Michael Grässl; Manuel Pühringer; Christophe Rauch; Hanna Küpper; Julia Maren Lüttgenau; Marion Pilz; Jonas Denecke; Melanie Haage-Brüning; Kathrin Mörtlbauer; Franziska Grünberg-Lemi; Marcus Deschauer; Caroline Deborah Stapf; Annekathrin Rödiger; Cornelia Voigt-Müller; Janina von der Gablentz; Beate Muschner; Ronny Nebelung; Alina Sapranidis; Magdalena Gosk - Tomek; Heike Losch; Maren Hackenberg; Alexandra Giese; Ursula Schneider Rosinger; Sybille Döhler; Maria Rehfeldt; Stephanie Schüssler; Stephan Wenninger; Barbara Ramadan; Svenja Neuhoff; Gudrun Zulehner; Rachel Fabian; Angela Kaindl; Johannes Flammer; Brigitte Brauner; Carmen Hollerauer; Tajana Brunckhorst; Anna Hotter; Gabriel Dworschak; Lisa Jung; Sabine Borowski; Timo Deba; Anne-Katrin Güttsches; Lisa Starke; Jürgen-Christoph von Kleist-Retzow; Ina Krahwinkler; Burkhard Gess; Lukas Neubauer; Michael Zemlin; Wolfgang Müller-Felber; Markus Blankenburg; Josefin Grabow; Philipp Hoth; Ilka Lehnert; Georg Friedrich Hoffmann; Arpad von Moers; Philipp Hoth; Petra Baum; Nina Rademacher; Ulf Hustedt; Bernhard Fasching; Jolanta Heinzinger; Magnus Ruf; Barbara Plecko; Marie-Luise Drax; Frank Kerling; Nadja Kaiser; Eleni Chatzizisi; Joenna Driemeyer; Meret Tabea Dreyer; Elke Pernegger; Isabell Cordts; Ilka Schneider; Benjamin Ilse; Sara-Maria Simon; Bernd Friedrich; Kurt Wollinsky; Janina Gburek-Augustat; Franz-Philipp Brändle; Anna Wiesenhofer; Iris Marquardt; Clemens Schächter; Christine Mauz; Christina Knellwolf; Maren Freigang; Raphael Seebröker; Simone Thiele; Melina Schlag; Jakob Rath; Margrit Koglin; Lisa Valerie Bitzan; Christine Leypold; Florian Junge; Katrin Schüler; Franziska Busch; Sabrina Geissler; Heike de Vries; Katja Köbbing; Ute Weyen; Ioanna Angeli; Barbara Plecko; Kerstin Böcking; Moritz Tacke; Michael Schroth; Frauke Ehrhardt; Monika Meyer; Nicole Claus; Stefan Kölker; Kathrin Bühner; Monika Meyer; Jennifer Rolack; Britta Holtkamp; Inka Brandes; Jakob Rath; Rahel Schuler; Elisabeth Steiner; Martin Drechsel; Dorothee Fütterer; Patricia Müller; Paula Steffens; Ulrike Wolf; Tanja Neumair; Marianne Rösner; Anna Katharina Koelsch; Almut Fritsch; Eva Jung; Clemens Runge; Kornelia Kreiser; Andreas Merkenschlager; Susanne Grinzinger; Yvonne Lechner; Renate Peters; Max Behrens; Adrian Tassoni; Zylfije Dibrani; Marian Kollaske; Laura Stimmer; Jaquelina Lipka; Martin Krenn; Martina Delitz; Alexandra Wagner; Katja D’Amico-Hofmann; Anna Resch; Lisa Allenstein; Stephanie Molitor; Peter Huppke; Andor Horváth; Jakob Wefers; Julia Turowski; Sabine Hettrich; Iris Hannibal; Lutz Dondit; Aglaia Lütjens; Stefan Kappel; Sandy Förster; Sabine Specht; Katharin Müller-Kaempffer; Stefan Kappel; Barbara Andres; Krisitin Kook; Martin Krenn; Andrea Hackemer; Doris Roland-Schäfer; Ingo Conze; Deike Weiss; Mario Müller; Anna Elmecker; Petra Rau; Thomas Kendzierski; Uta Smesny; Nikolai Jung; Astri Fromm; Johannes Dorst; Henriette Kiep; Christian Rauscher; Katia Vettori; Tobias Linden; Tim Kampowski; Anja Boegner; Eva Malm; Fritz Zimprich; Barbara Schwegmann; Vladimir Dukic; Rike Remmert; Christa Bretschneider; Barbara Fiedler; Corinna Rademacher; Loreen Plugge; Sybille Stephan-Lutter; Lena Manssen; Eva Wendel; Martina Neininger; Anja Meenken; Moritz Niesert; Annika Meenken; Nina Schlaghecke; Meike Kahrs; Fritz Zimprich; Lena Ruß; Eva Jansen; Heidi Starke; Jörg Tiedemann; Verena Angermair; Vincent Gmeiner; Sebastian Plutz; Anna-Lena Fleischer; Sabine Wider; Kristin Loyal; Victoria Göbel; Alexander Bobe; Anna-Maria Gaese; Katharina Längle; Birgitt Moed; Franziska Wenzel; Katharina Wagner; Peter Reilich; Thomas Dörnen; Corinna Stoltenburg; Hanna Reinke-Hermer; Sindy Becker; Eva Johann to Settel; Karsten Krause; Christine Sprengart; Birgit Warken-Madelung; Daniela Banholzer; Katja Koch; Urania Kotzaeridou; Katja Koch; Jana Barluschke; Kirsten Fündeling-Tielitz; Gudrun Zulehner; Katharina Unverfehrt; Esther Maihöfer; Ricarda von Heynitz; Max Obenauf; Nadin Fedtke; Juliane Hug; Mohammad Kalaf; Julia Seifert; Ramona Menzel; Mirjam Spitzinger; Anna Hüpper; Shalin Nerkamp; Lukas Nalbach; Beate Schlotter-Weigel; Zoé Messerli; Anja Müller; Geerthe Margriet Balk; Irene Lange; Nicole Groß; Annika Roser-Unruh; Elke Preisendanz; Jessica Ribke; Annegret Hoevel; Jessica Ribke; Corinna Doege; Daniel Bormann; Heidrun Krämer-Best; Birgit Meßmer; Barbara Vogel; David Strube; Saskia Beyer; Matthias Eckenweiler; Maj-Britt Bartels; Aneliya Simeonova Eroglu; Elena Gantschew; Elke Tiefenthaler; Nora Maier; Janina Fürup; David Leibensperger; Laura Kahle; Sibylle Vogt; Sabine Stein
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Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland—part I—systematic literature review and existing evidence德国、奥地利和瑞士关于Onasemnogene Abeparvovec治疗脊髓性肌萎缩基因疗法的德尔菲共识——第一部分——系统文献综述与现有证据
err2025-11-24
err0
errOAAI
errClaudia Weiß; Katharina Vill; Matthias Baumann; Günther Bernert; Astrid Blaschek; Astrid Eisenkölbl; Marina Flotats-Bastardas; Johannes Friese; Claudia Ganter; Klaus Goldhahn; Andreas Hahn; Maja von der Hagen; Hans Hartmann; Oswald Hasselmann; Veronka Horber; Ralf A Husain; Sabine Illsinger; David Jacquier; Jessika Johannsen; Cornelia Köhler; Heike Kölbel; Michael Kolodzig; Andrea Klein; Astrid Pechmann; Arpad von Moers; Wolfgang Müller-Felber; Christian Rauscher; Ulrike Schara-Schmidt; Gudrun Schreiber; Oliver Schwartz; Joachim Sproß; Georg M Stettner; Corinna Stoltenburg; Eva Stumpe; Regina Trollmann; Gert Wiegand; Ekkehard Wilichowski; Janbernd Kirschner; Andreas Ziegler
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Respiratory function in 192 adult patients with spinal muscular atrophy (SMA) treated with nusinersen – a multicenter observational study192例接受nusinersen治疗的脊髓性肌萎缩症(SMA)成年患者的呼吸功能——一项多中心观察性研究
err2025-09-29
err0
errOAAI
errClaudia D. Wurster; Benjamin Stolte; Tobias Kessler; Maren Freigang; Bogdan Bjelica; Benjamin Ilse; Jan C. Koch; Isabell Cordts; Alexander Mensch; Daniel Zeller; Zeljko Uzelac; Georges Sam; Hanna Sophie Lapp; Camilla Wohnrade; Annekathrin Rödiger; Mohamad Tareq Muhandes; Ilka Schneider; Julia Bellut; Julia Nentwich; Johannes Dorst; Joachim Schuster; Olivia Schreiber-Katz; Alma Osmanovic; Andreas Totzeck; Andreas Thimm; Robert Steinbach; Julian Grosskreutz; Christoph Kleinschnitz; Albert C. Ludolph; Marcus Deschauer; Janbernd Kirschner; Jens Dreyhaupt; Kurt Wollinsky; Susanne Petri; Markus Weiler; Tim Hagenacker; René Günther
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Phenotypic intrafamilial variability of 5q-associated spinal muscular atrophy: A systematic multicentre sibling study5q相关脊髓性肌萎缩症的表型家族内变异性:一项系统性多中心同胞研究
err2025-09-12
err0
PREAI
errBenedikt Becker; Isabell Cordts; Jutta Becker; Rene Günther; Matthias Baumann; Günther Bernert; Astrid Eisenkölbl; Barbara Fiedler; Marina Flotats-Bastardas; Martin Fleger; Tim Hagenacker; Andreas Hahn; Elke Hobbiebrunken; Andrea Bevot; Jörg Jahnel; Jessika Johannsen; Christoph Kamm; Jan Christoph Koch; Cornelia Köhler; Heike Kölbel; Wolfgang Müller-Felber; Christoph Neuwirth; Barbara Plecko; Christian Stadler; Martin Smitka; Arpad Von Moers; Regina Trollmann; Markus Weiler; Andreas Ziegler; Susanne Goldbach; Kristina Probst-Schendzielorz; Hanns Lochmüller; Ulrike Schara-Schmidt; Maggie C Walter; Janbernd Kirschner; Brunhilde Wirth; Astrid Pechmann; Marcus Deschauer
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A Statistical Approach to Latent Dynamic Modeling with Differential Equations基于微分方程的潜在动态建模的统计方法
err2025-09-01
err0
PREAI
errHackenberg, Maren; Pechmann, Astrid; Kreutz, Clemens; Kirschner, Janbernd; Binder, Harald
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Physiotherapeutic management of patients with SMA: A questionnaire-based online survey among physiotherapists within the SMArtCARE network脊髓性肌萎缩症患者的物理治疗管理:SMArtCARE网络内物理治疗师参与的基于问卷的在线调查
err2025-08-19
err0
errOAAI
errSibylle Vogt; Cornelia Voigt-Müller; Heidi Rochau-Trumpp; Eva Malm; Doris Roland-Schäfer; Gerda Roetmann; Judith van Vugt; Sabine Stein; Janbernd Kirschner; Astrid Pechmann
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TREAT: systematic and inclusive selection process of genes for genomic newborn screening as part of the Screen4Care projectTREAT:Screen4Care项目框架下的基因组新生儿筛查基因的系统性及包容性选择流程
err2025-05-15
err0
errOAAI
errSaier, Christina; Sansen, Stefaan; Berghout, Joanne; Freyler, Kathrin; Einhorn, Moshe; Einhorn, Yaron; Matalonga, Leslie; Beltran, Sergi; Novelli, Antonio; Selvatici, Rita; Fortunato, Fernanda; Montanari, Silvia; Martinez-Fresno, Maria; Gumus, Gulcin; Agolini, Emanuele; Garnier, Nicolas; Ferlini, Alessandra; Bertini, Enrico; Kirschner, Janbernd
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Characterization of severe COL6-related dystrophy due to the recurrent variant COL6A1 c.930+189C>TCOL6相关严重型肌营养不良的表征:由复发性变异COL6A1 c.930+189C>T引起
errBrain
IF11.7
err2025-04-03
err0
errOAAI
errA Reghan Foley; Véronique Bolduc; Fady Guirguis; Sandra Donkervoort; Ying Hu; Rotem Orbach; Riley M McCarty; Apurva Sarathy; Gina Norato; Beryl B Cummings; Monkol Lek; Anna Sarkozy; Russell J Butterfield; Janbernd Kirschner; Andrés Nascimento; Daniel Natera-de Benito; Susana Quijano-Roy; Tanya Stojkovic; Luciano Merlini; Giacomo Comi; Monique Ryan; Denise McDonald; Pinki Munot; Grace Yoon; Edward Leung; Erika Finanger; Meganne E Leach; James Collins; Cuixia Tian; Payam Mohassel; Sarah B Neuhaus; Dimah Saade; Benjamin T Cocanougher; Mary-Lynn Chu; Mena Scavina; Carla Grosmann; Randal Richardson; Brian D Kossak; Sidney M Gospe; Vikram Bhise; Gita Taurina; Baiba Lace; Monica Troncoso; Mordechai Shohat; Adel Shalata; Sophelia H S Chan; Manu Jokela; Johanna Palmio; Göknur Haliloğlu; Cristina Jou; Corine Gartioux; Herimela Solomon-Degefa; Carolin D Freiburg; Alvise Schiavinato; Haiyan Zhou; Sara Aguti; Yoram Nevo; Ichizo Nishino; Cecilia Jimenez-Mallebrera; Shireen R Lamandé; Valérie Allamand; Francesca Gualandi; Alessandra Ferlini; Daniel G MacArthur; Steve D Wilton; Raimund Wagener; Enrico Bertini; Francesco Muntoni; Carsten G Bönnemann
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Efficacy fi cacy 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
err2024-12-01
err0
errOAAI
errWeiss, Claudia; Becker, Lena-Luise; Friese, Johannes; Blaschek, Astrid; Hahn, Andreas; Illsinger, Sabine; Schwartz, Oliver; Bernert, Guenther; von der Hagen, Maja; Husain, Ralf A.; Goldhahn, Klaus; Kirschner, Janbernd; Pechmann, Astrid; Flotats-Bastardas, Marina; Schreiber, Gudrun; Schara, Ulrike; Plecko, Barbara; Trollmann, Regina; Horber, Veronka; Wilichowski, Ekkehard; Baumann, Matthias; Klein, Andrea; Eisenkoelbl, Astrid; Koehler, Cornelia; Stettner, Georg M.; Cirak, Sebahattin; Hasselmann, Oswald; Kaindl, Angela M.; Garbade, Sven F.; Johannsen, Jessika; Ziegler, Andreas
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Clinical Effectiveness of Newborn Screening for Spinal Muscular Atrophy: A Nonrandomized Controlled Trial
err2024-12-01
err0
PREAI
errSchwartz, Oliver; Vill, Katharina; Pfaffenlehner, Michelle; Behrens, Max; Weiss, Claudia; Johannsen, Jessika; Friese, Johannes; Hahn, Andreas; Ziegler, Andreas; Illsinger, Sabine; Smitka, Martin; von Moers, Arpad; Koelbel, Heike; Schreiber, Gudrun; Kaiser, Nadja; Wilichowski, Ekkehard; Flotats-Bastardas, Marina; Husain, Ralf A.; Baumann, Matthias; Koehler, Cornelia; Trollmann, Regina; Schwerin-Nagel, Annette; Eisenkoelbl, Astrid; Schimmel, Mareike; Fleger, Martin; Kauffmann, Birgit; Wiegand, Gert; Baumgartner, Manuela; Rauscher, Christian; Cirak, Sebahattin; Glaeser, Dieter; Bernert, Guenther; Hagenacker, Tim; Goldbach, Susanne; Probst-Schendzielorz, Kristina; Lochmueller, Hanns; Mueller-Felber, Wolfgang; Schara-Schmidt, Ulrike; Walter, Maggie C.; Kirschner, Janbernd; Pechmann, Astrid; SMARTCARE Study Group
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Concerns About Clinical Effectiveness of Newborn Screening for Spinal Muscular Atrophy-Reply
err2024-09-01
err0
PREAI
errSchwartz, Oliver; Pechmann, Astrid; Kirschner, Janbernd
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Clinical Effectiveness of Newborn Screening for Spinal Muscular Atrophy
err2024-06-01
err12
PREAI
errSchwartz, Oliver; Vill, Katharina; Pfaffenlehner, Michelle; Behrens, Max; Weiss, Claudia; Johannsen, Jessika; Friese, Johannes; Hahn, Andreas; Ziegler, Andreas; Illsinger, Sabine; Smitka, Martin; von Moers, Arpad; Koelbel, Heike; Schreiber, Gudrun; Kaiser, Nadja; Wilichowski, Ekkehard; Flotats-Bastardas, Marina; Husain, Ralf A.; Baumann, Matthias; Koehler, Cornelia; Trollmann, Regina; Schwerin-Nagel, Annette; Eisenkoelbl, Astrid; Schimmel, Mareike; Fleger, Martin; Kauffmann, Birgit; Wiegand, Gert; Baumgartner, Manuela; Rauscher, Christian; Cirak, Sebahattin; Glaeser, Dieter; Bernert, Guenther; Hagenacker, Tim; Goldbach, Susanne; Probst-Schendzielorz, Kristina; Lochmueller, Hanns; Mueller-Felber, Wolfgang; Schara-Schmidt, Ulrike; Walter, Maggie C.; Kirschner, Janbernd; Pechmann, Astrid
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Increased Diagnostic Yield by Reanalysis of Whole Exome Sequencing Data in Mitochondrial Disease
err2024-05-13
err0
errOAAI
errOlimpio, Catarina; Paramonov, Ida; Matalonga, Leslie; Laurie, Steven; Schon, Katherine; Polavarapu, Kiran; Kirschner, Janbernd; Schara-Schmidt, Ulrike; Lochmueller, Hanns; Chinnery, Patrick F.; Horvath, Rita
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Predictors of Loss of Ambulation in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis
err2024-04-22
err5
errOAAI
errLandfeldt, E.; Aleman, A.; Abner, S.; Zhang, R.; Werner, C.; Tomazos, I.; Ferizovic, N.; Lochmueller, H.; Kirschner, J.
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Long-term efficacy and safety of nusinersen in adults with 5q spinal muscular atrophy: a prospective European multinational observational study
err2024-04-01
err9
errOAAI
errGuenther, Rene; 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; Roediger, Annekathrin; Bellut, Julia; Nentwich, Julia; Zeller, Daniel; Muhandes, Mohamad Tareq; Baum, Tobias; Koch, Jan Christoph; 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; Tuerk, Matthias; Schimmel, Mareike; Bernert, Guenther; Martin, Pascal; Rauscher, Christian; Hoerste, Gerd Meyer zu; Baum, Petra; Loescher, Wolfgang; Flotats-Bastardas, Marina; Koehler, Cornelia; Probst-Schendzielorz, Kristina; Goldbach, Susanne; Schara-Schmidt, Ulrike; Mueller-Felber, Wolfgang; Lochmueller, Hanns; von Velsen, Otgonzul; Kleinschnitz, Christoph; Ludolph, Albert C.; Hagenacker, Tim
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Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry用Onasemnogene Abeparvovec单药治疗的脊髓性肌萎缩症患者的真实世界结果: 来自RESTORE注册中心的发现
err2024-01-18
err20
errOAAI
errServais, Laurent; Day, John W.; De Vivo, Darryl C.; Kirschner, Janbernd; Mercuri, Eugenio; Muntoni, Francesco; Proud, Crystal M.; Shieh, Perry B.; Tizzano, Eduardo F.; Quijano-Roy, Susana; Desguerre, Isabelle; Saito, Kayoko; Faulkner, Eric; Benguerba, Kamal M.; Raju, Dheeraj; LaMarca, Nicole; Sun, Rui; Anderson, Frederick A.; Finkel, Richard S.
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