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Long-term CSF responses in adult patients with spinal muscular atrophy type 2 or 3 on treatment with nusinersen by Cebulla, Gina (Author) , Hai, Ling (Author) , Warnken, Uwe (Author) , Güngör, Cansu (Author) , Hoffmann, Dirk C. (Author) , Korporal-Kuhnke, Mirjam (Author) , Wildemann, Brigitte (Author) , Wick, Wolfgang (Author) , Keßler, Tobias (Author) , Weiler, Markus (Author) ,

• Verlag, kostenfrei, Volltext

Cognitive function in SMA patients with 2 or 3 SMN2 copies treated with SMN-modifying or gene addition therapy during the first year of life by Steffens, Paula (Author) , Weiss, Deike (Author) , Perez, Anna (Author) , Appel, Manuel (Author) , Weber, Philipp (Author) , Weiss, Claudia (Author) , Stoltenburg, Corinna (Author) , Ehinger, Ute (Author) , von der Hagen, Maja (Author) , Schallner, Jens (Author) , Claussen, Birte (Author) , Lode, Ilka (Author) , Hahn, Andreas (Author) , Schuler, Rahel (Author) , Ruß, Lena (Author) , Ziegler, Andreas (Author) , Denecke, Jonas (Author) , Johannsen, Jessika (Author) ,

• Verlag, kostenfrei, Volltext
• Verlag, kostenfrei, Volltext

European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy by Kirschner, Janbernd (Author) , Butoianu, Nina (Author) , Goemans, Nathalie (Author) , Haberlova, Jana (Author) , Kostera-Pruszczyk, Anna (Author) , Mercuri, Eugenio (Author) , van der Pol, W. Ludo (Author) , Quijano-Roy, Susana (Author) , Sejersen, Thomas (Author) , Tizzano, Eduardo F. (Author) , Ziegler, Andreas (Author) , Servais, Laurent (Author) , Muntoni, Francesco (Author) ,

• Verlag, lizenzpflichtig, Volltext
• Verlag, lizenzpflichtig, Volltext