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

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; Muller-Felber, Wolfgang; Vill, Katharina; Blaschek, Astrid; Smitka, Martin; van der Stam, Lieske; Weiss, Katja; Winter, Benedikt; Goldhahn, Klaus; Plecko, Barbara; Horber, Veronka; Bernert, Gunther; Husain, Ralf A.; Rauscher, Christian; Trollmann, Regina; Garbade, Sven F.; Hahn, Andreas; von der Hagen, Maja; Kaindl, Angela M.

dc.contributor.author	Horber, Veronka
dc.date.accessioned	2023-02-28T11:00:36Z
dc.date.available	2023-02-28T11:00:36Z
dc.date.issued	2022
dc.identifier.issn	2352-4642
dc.identifier.uri	http://hdl.handle.net/10900/137105
dc.language.iso	en	de_DE
dc.publisher	Elsevier Sci Ltd	de_DE
dc.relation.uri	http://dx.doi.org/10.1016/S2352-4642(21)00287-X	de_DE
dc.subject.ddc	610	de_DE
dc.title	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	de_DE
dc.type	Article	de_DE
utue.quellen.id	20220728000000_02692
utue.publikation.seiten	17-27	de_DE
utue.personen.roh	Weiss, Claudia
utue.personen.roh	Ziegler, Andreas
utue.personen.roh	Becker, Lena-Luise
utue.personen.roh	Johannsen, Jessika
utue.personen.roh	Brennenstuhl, Heiko
utue.personen.roh	Schreiber, Gudrun
utue.personen.roh	Flotats-Bastardas, Marina
utue.personen.roh	Stoltenburg, Corinna
utue.personen.roh	Hartmann, Hans
utue.personen.roh	Illsinger, Sabine
utue.personen.roh	Denecke, Jonas
utue.personen.roh	Pechmann, Astrid
utue.personen.roh	Muller-Felber, Wolfgang
utue.personen.roh	Vill, Katharina
utue.personen.roh	Blaschek, Astrid
utue.personen.roh	Smitka, Martin
utue.personen.roh	van der Stam, Lieske
utue.personen.roh	Weiss, Katja
utue.personen.roh	Winter, Benedikt
utue.personen.roh	Goldhahn, Klaus
utue.personen.roh	Plecko, Barbara
utue.personen.roh	Horber, Veronka
utue.personen.roh	Bernert, Gunther
utue.personen.roh	Husain, Ralf A.
utue.personen.roh	Rauscher, Christian
utue.personen.roh	Trollmann, Regina
utue.personen.roh	Garbade, Sven F.
utue.personen.roh	Hahn, Andreas
utue.personen.roh	von der Hagen, Maja
utue.personen.roh	Kaindl, Angela M.
dcterms.isPartOf.ZSTitelID	Lancet Child & Adolescent Health	de_DE
dcterms.isPartOf.ZS-Issue	1	de_DE
dcterms.isPartOf.ZS-Volume	6	de_DE
utue.fakultaet	04 Medizinische Fakultät	de_DE