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

Weiss, 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

dc.contributor.author	Horber, Veronka
dc.date.accessioned	2025-05-27T06:46:14Z
dc.date.available	2025-05-27T06:46:14Z
dc.date.issued	2024
dc.identifier.issn	2666-7762
dc.identifier.uri	http://hdl.handle.net/10900/165942
dc.language.iso	en	de_DE
dc.publisher	Amsterdam : Elsevier	de_DE
dc.relation.uri	http://dx.doi.org/10.1016/j.lanepe.2024.101092	de_DE
dc.subject.ddc	300	de_DE
dc.subject.ddc	610	de_DE
dc.title	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	de_DE
dc.type	Article	de_DE
utue.quellen.id	20250203000000_00976
utue.personen.roh	Weiss, Claudia
utue.personen.roh	Becker, Lena-Luise
utue.personen.roh	Friese, Johannes
utue.personen.roh	Blaschek, Astrid
utue.personen.roh	Hahn, Andreas
utue.personen.roh	Illsinger, Sabine
utue.personen.roh	Schwartz, Oliver
utue.personen.roh	Bernert, Guenther
utue.personen.roh	von der Hagen, Maja
utue.personen.roh	Husain, Ralf A.
utue.personen.roh	Goldhahn, Klaus
utue.personen.roh	Kirschner, Janbernd
utue.personen.roh	Pechmann, Astrid
utue.personen.roh	Flotats-Bastardas, Marina
utue.personen.roh	Schreiber, Gudrun
utue.personen.roh	Schara, Ulrike
utue.personen.roh	Plecko, Barbara
utue.personen.roh	Trollmann, Regina
utue.personen.roh	Horber, Veronka
utue.personen.roh	Wilichowski, Ekkehard
utue.personen.roh	Baumann, Matthias
utue.personen.roh	Klein, Andrea
utue.personen.roh	Eisenkoelbl, Astrid
utue.personen.roh	Koehler, Cornelia
utue.personen.roh	Stettner, Georg M.
utue.personen.roh	Cirak, Sebahattin
utue.personen.roh	Hasselmann, Oswald
utue.personen.roh	Kaindl, Angela M.
utue.personen.roh	Garbade, Sven F.
utue.personen.roh	Johannsen, Jessika
utue.personen.roh	Ziegler, Andreas
dcterms.isPartOf.ZSTitelID	Lancet Regional Health - Europe	de_DE
dcterms.isPartOf.ZS-Issue	Article 101092	de_DE
dcterms.isPartOf.ZS-Volume	47	de_DE
utue.fakultaet	04 Medizinische Fakultät	de_DE