Efficacy 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

C Weiß; LL Becker; J Friese; A Blaschek; A Hahn; S Illsinger; O Schwartz; G Bernert; MV Hagen; RA Husain; K Goldhahn; J Kirschner; A Pechmann; M Flotats-Bastardas; G Schreiber; U Schara; B Plecko; R Trollmann; V Horber; E Wilichowski; M Baumann; A Klein; Astrid Eisenkölbl; C Köhler; GM Stettner; S Cirak; O Hasselmann; AM Kaindl; SF Garbade; J Johannsen; A. Ziegler; SMArtCARE and Swiss-Reg-NMD study group

doi:10.1016/j.lanepe.2024.101092

Efficacy 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

C Weiß
, LL Becker
, J Friese
, A Blaschek
, A Hahn
, S Illsinger
, O Schwartz
, G Bernert
, MV Hagen
, RA Husain
, K Goldhahn
, J Kirschner
, A Pechmann
, M Flotats-Bastardas
, G Schreiber
, U Schara
, B Plecko
, R Trollmann
, V Horber
, E Wilichowski

M Baumann, A Klein, Astrid Eisenkölbl, C Köhler, GM Stettner, S Cirak, O Hasselmann, AM Kaindl, SF Garbade, J Johannsen, A. Ziegler, SMArtCARE and Swiss-Reg-NMD study group

Department of Pediatrics and Adolescent Medicine

Research output: Contribution to journal › Article › peer-review

Original language	English
Article number	101092
Number of pages	1
Journal	The Lancet Regional Health - Europe
Volume	47
DOIs	https://doi.org/10.1016/j.lanepe.2024.101092
Publication status	Published - Dec 2024

Fields of science

302035 Paediatrics and adolescent medicine

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10.1016/j.lanepe.2024.101092

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Weiß, C., Becker, LL., Friese, J., Blaschek, A., Hahn, A., Illsinger, S., Schwartz, O., Bernert, G., Hagen, MV., Husain, RA., Goldhahn, K., Kirschner, J., Pechmann, A., Flotats-Bastardas, M., Schreiber, G., Schara, U., Plecko, B., Trollmann, R., Horber, V., ... SMArtCARE and Swiss-Reg-NMD study group (2024). Efficacy 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. The Lancet Regional Health - Europe, 47, Article 101092. https://doi.org/10.1016/j.lanepe.2024.101092

@article{86da3c88d6244317944b08cd13ff028d,

title = "Efficacy 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",

author = "C Wei{\ss} and LL Becker and J Friese and A Blaschek and A Hahn and S Illsinger and O Schwartz and G Bernert and MV Hagen and RA Husain and K Goldhahn and J Kirschner and A Pechmann and M Flotats-Bastardas and G Schreiber and U Schara and B Plecko and R Trollmann and V Horber and E Wilichowski and M Baumann and A Klein and Astrid Eisenk{\"o}lbl and C K{\"o}hler and GM Stettner and S Cirak and O Hasselmann and AM Kaindl and SF Garbade and J Johannsen and A. Ziegler and \{SMArtCARE and Swiss-Reg-NMD study group\}",

year = "2024",

month = dec,

doi = "10.1016/j.lanepe.2024.101092",

language = "English",

volume = "47",

journal = "The Lancet Regional Health - Europe",

issn = "2666-7762",

}

Weiß, C, Becker, LL, Friese, J, Blaschek, A, Hahn, A, Illsinger, S, Schwartz, O, Bernert, G, Hagen, MV, Husain, RA, Goldhahn, K, Kirschner, J, Pechmann, A, Flotats-Bastardas, M, Schreiber, G, Schara, U, Plecko, B, Trollmann, R, Horber, V, Wilichowski, E, Baumann, M, Klein, A, Eisenkölbl, A, Köhler, C, Stettner, GM, Cirak, S, Hasselmann, O, Kaindl, AM, Garbade, SF, Johannsen, J, Ziegler, A & SMArtCARE and Swiss-Reg-NMD study group 2024, 'Efficacy 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', The Lancet Regional Health - Europe, vol. 47, 101092. https://doi.org/10.1016/j.lanepe.2024.101092

TY - JOUR

T1 - Efficacy 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

AU - Weiß, C

AU - Becker, LL

AU - Friese, J

AU - Blaschek, A

AU - Hahn, A

AU - Illsinger, S

AU - Schwartz, O

AU - Bernert, G

AU - Hagen, MV

AU - Husain, RA

AU - Goldhahn, K

AU - Kirschner, J

AU - Pechmann, A

AU - Flotats-Bastardas, M

AU - Schreiber, G

AU - Schara, U

AU - Plecko, B

AU - Trollmann, R

AU - Horber, V

AU - Wilichowski, E

AU - Baumann, M

AU - Klein, A

AU - Eisenkölbl, Astrid

AU - Köhler, C

AU - Stettner, GM

AU - Cirak, S

AU - Hasselmann, O

AU - Kaindl, AM

AU - Garbade, SF

AU - Johannsen, J

AU - Ziegler, A.

AU - SMArtCARE and Swiss-Reg-NMD study group, null

PY - 2024/12

Y1 - 2024/12

UR - https://www.scopus.com/pages/publications/85205675214

U2 - 10.1016/j.lanepe.2024.101092

DO - 10.1016/j.lanepe.2024.101092

M3 - Article

C2 - 39434961

SN - 2666-7762

VL - 47

JO - The Lancet Regional Health - Europe

JF - The Lancet Regional Health - Europe

M1 - 101092

ER -

Efficacy 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

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