Chiriboga, Claudia A. http://orcid.org/0000-0001-6088-4889
Bruno, Claudio
Duong, Tina http://orcid.org/0000-0003-3177-4722
Fischer, Dirk
Mercuri, Eugenio http://orcid.org/0000-0002-9851-5365
Kirschner, Janbernd http://orcid.org/0000-0003-1618-7386
Kostera-Pruszczyk, Anna http://orcid.org/0000-0002-7309-1914
Jaber, Birgit
Gorni, Ksenija http://orcid.org/0000-0002-7510-1127
Kletzl, Heidemarie http://orcid.org/0000-0001-8129-2196
Carruthers, Imogen
Martin, Carmen
Scalco, Renata S. http://orcid.org/0000-0002-5400-562X
Fontoura, Paulo http://orcid.org/0000-0002-3883-257X
Muntoni, Francesco http://orcid.org/0000-0002-9102-5232
,
Funding for this research was provided by:
F. Hoffmann-La Roche
Article History
Received: 26 January 2024
Revised: 8 March 2024
Accepted: 8 March 2024
First Online: 11 May 2024
Declarations
:
: CAC has taken part in advisory boards for AveXis/Novartis, Biogen, PTC Therapeutics, Genentech, and F. Hoffmann-La Roche Ltd, received educational speaker fees from Biogen, F. Hoffmann-La Roche, and Genentech and received research support grants from AveXis/Novartis, Biogen, F. Hoffmann-La Roche Ltd, and the National Institutes of Health. CB has received honoraria for scientific advisory boards from Biogen, Novartis, F. Hoffmann-La Roche Ltd, and Sarepta Therapeutics, and has received research support grants from Biogen. TD has received honoraria for scientific advisory boards or consultancy from Biogen, Novartis, F. Hoffmann-La Roche Ltd, Genentech, Pfizer, Sarepta Therapeutics, Audentes, Astellas, and Dyne. DF has received honoraria for scientific advisory boards or consultancy from F. Hoffmann-La Roche Ltd. EM has received honoraria for scientific advisory boards and educational speaker fees from Sarepta Therapeutics, Santhera, PTC Therapeutics, Pfizer, F. Hoffmann-La Roche Ltd, Biogen, Avexis, Novartis, Scholar Rock, and Cytokinetics, and has received research support grants from Biogen. JK has received honoraria for scientific advisory boards from Biogen, Novartis, PTC Therapeutics, Pfizer, F. Hoffmann-La Roche Ltd, and Sarepta Therapeutics and reports grants from Biogen, Novartis, and F. Hoffmann-La Roche Ltd. AKP has received compensation for participation at symposia, lectures, and scientific advisory boards from Biogen, F. Hoffmann-La Roche Ltd, AveXis/Novartis, and PTC Therapeutics. She has also received institutional grant support from Biogen and is an investigator in SMA trials sponsored by F. Hoffmann-La Roche Ltd. BJ, KG, HK, CM, RSS, and PF are employees and shareholders of F. Hoffmann-La Roche Ltd, Basel, Switzerland. FM is member of the Pfizer Rare Disease Advisory Board and of Dyne Therapeutic SAB.
: This trial was approved by an ethics committee at each study site and was conducted in accordance with Good Clinical Practice guidelines and with the World Medical Association Declaration of Helsinki.
: Written informed consent was obtained from the patient or from the patients’ legally authorized representative before participating in the study.