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Author/Creator Müller-Felber, Wolfgang

1. Gene replacement therapy with onasemnogene abeparvovec in children with spinal muscular atrophy aged 24 months or younger and bodyweight up to 15 kg: an observational cohort study. (January 2022)

Authors:
Weiß, Claudia; Ziegler, Andreas; Becker, Lena-Luise; Johannsen, Jessika; Brennenstuhl, Heiko; Schreiber, Gudrun; Flotats-Bastardas, Marina; Stoltenburg, Corinna; Hartmann, Hans; Illsinger, Sabine; Denecke, Jonas; Pechmann, Astrid; Müller-Felber, Wolfgang; Vill, Katharina; Blaschek, Astrid; Smitka...
Journal:
Lancet
Issue:
Volume 6:Number 1(2022)
Page Start:
17
Record Type:
Journal Article
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Available online (eLD content is only available in our Reading Rooms) ↗

3. Prevalence of Pompe disease in 3, 076 patients with hyperCKemia and limb-girdle muscular weakness. (19th July 2016)

Authors:
Lukacs, Zoltan; Nieves Cobos, Paulina; Wenninger, Stephan; Willis, Tracey A.; Guglieri, Michela; Roberts, Marc; Quinlivan, Rosaline; Hilton-Jones, David; Evangelista, Teresinha; Zierz, Stephan; Schlotter-Weigel, Beate; Walter, Maggie C.; Reilich, Peter; Klopstock, Thomas; Deschauer, Marcus; Strau...
Journal:
Neurology
Issue:
Volume 87:Number 3(2016)
Page Start:
Record Type:
Journal Article
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Available online (eLD content is only available in our Reading Rooms) ↗