Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry c...
Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022 interim analysis
About this item
Full title
Author / Creator
STRIDE and CINRG DNHS investigators , the STRIDE and CINRG DNHS investigators , Mercuri, Eugenio , Osorio, Andrés Nascimento , Muntoni, Francesco , Buccella, Filippo , Desguerre, Isabelle , Kirschner, Janbernd , Tulinius, Már , de Resende, Maria Bernadete Dutra , Morgenroth, Lauren P. , Gordish-Dressman, Heather , Johnson, Shelley , Kristensen, Allan , Werner, Christian , Trifillis, Panayiota , Henricson, Erik K. and McDonald, Craig M.
Publisher
Berlin/Heidelberg: Springer Berlin Heidelberg
Journal title
Language
English
Formats
Publication information
Publisher
Berlin/Heidelberg: Springer Berlin Heidelberg
Subjects
More information
Scope and Contents
Contents
Objective
Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, international, multicenter registry of real-world ataluren use in individuals with nonsense mutation Duchenne muscular dystrophy (nmDMD) in clinical practice. This updated interim report (data cut-off: January 31, 2022), describes STRIDE...
Alternative Titles
Full title
Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022 interim analysis
Authors, Artists and Contributors
Author / Creator
the STRIDE and CINRG DNHS investigators
Mercuri, Eugenio
Osorio, Andrés Nascimento
Muntoni, Francesco
Buccella, Filippo
Desguerre, Isabelle
Kirschner, Janbernd
Tulinius, Már
de Resende, Maria Bernadete Dutra
Morgenroth, Lauren P.
Gordish-Dressman, Heather
Johnson, Shelley
Kristensen, Allan
Werner, Christian
Trifillis, Panayiota
Henricson, Erik K.
McDonald, Craig M.
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_swepub_primary_oai_gup_ub_gu_se_327280
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_swepub_primary_oai_gup_ub_gu_se_327280
Other Identifiers
ISSN
0340-5354
E-ISSN
1432-1459
DOI
10.1007/s00415-023-11687-1
