Cances, Claude http://orcid.org/0000-0001-7386-2275
Vlodavets, Dmitry
Comi, Giacomo Pietro
Masson, Riccardo
Mazurkiewicz-Bełdzińska, Maria
Saito, Kayoko
Zanoteli, Edmar
Dodman, Angela
El-Khairi, Muna
Gorni, Ksenija
Gravestock, Isaac
Hoffart, Janine
Scalco, Renata S.
Darras, Basil T.
Alberti, Katia
Baranello, Giovanni
Barisic, Nina
Brolatti, Noemi
Bruno, Claudio
Cances, Claude
Comi, Giacomo Pietro
Darras, Basil T.
Deconinck, Nicolas
Vos, Elke
De Waele, Liesbeth
Dodman, Angela
Dosi, Claudia
El-Khairi, Muna
Engelbrekt, Amanda
Goemans, Nathalie
Gorni, Ksenija
Govoni, Alessandra
Gravestock, Isaac
Haginoya, Kazuhiro
Hoffart, Janine
Kotulska-Jozwiak, Katarzyna
Goff, Laure Le
Levine, Alexis
Manel, Saidi
Masson, Riccardo
Mastella, Chiara
Mauri, Eleonora
Mazurkiewicz-Bełdzińska, Maria
Meneri, Megi
Moroni, Isabella
Pierzchlewicz, Katarzyna
Portefaix, Aurelie
Prufer, Alexandra
Rauso, Myriam
Saito, Kayoko
Scalco, Renata S.
Schembri, Veronica
Sicolo, Mariangela
Tahon, Valentine
Tomas, Josipa
Vincent-Genod, Dominique
Vlodavets, Dmitry
Vuillerot, Carole
Yotsumata, Kazuyuki
Zanoteli, Edmar
,
Funding for this research was provided by:
F. Hoffmann-La Roche
Article History
Received: 8 March 2022
Accepted: 17 July 2022
First Online: 29 July 2022
Declarations
:
: The study protocol was approved by an institutional review board/ethics committee at each study site and the study was conducted in accordance with Good Clinical Practice guidelines and the laws and regulations of each country in which the research was conducted. When required by local regulations, written informed consent was provided by parents or caregivers of the patients.
: Not applicable.
: CC is a site principal investigator for Biogen and F. Hoffmann-La Roche Ltd clinical trials, and has received advisory fees from Novartis TG. VD and GPC have no conflicts of interest. RM has received fees from Biogen, F. Hoffmann-La Roche Ltd and Novartis Gene Therapies. MMB is a site principal investigator for Biogen and F. Hoffmann-La Roche Ltd clinical trials, and has received honoraria for advisory boards and speaker's fees from Biogen, F. Hoffmann-La Roche Ltd, and Novartis. KS is a site principal investigator for Biogen and Novartis Gene Therapies clinical trials, has received honoraria for advisory boards from Biogen, Novartis, and Roche/Chugai and speaker’s fees from Biogen and Novartis. IG and JH are employees of F. Hoffmann-La Roche Ltd. AD, MEK, KG and RSS are employees of, and hold shares in, F. Hoffmann-La Roche Ltd. BTD has received grants from Biogen, CureSMA, F. Hoffmann-La Roche Ltd, Fibrogen, Ionis Pharmaceuticals, U.S. National Institutes of Health/National Institute of Neurological Disorders and Stroke, PTC Therapeutics, Sarepta Pharmaceuticals, Slaney Family Fund for SMA, Spinal Muscular Atrophy Foundation, Summit and Working on Walking Fund; and is a board member for Amicus Inc., AveXis, Biogen, F. Hoffmann-La Roche Ltd grants, Genentech, Sarepta Pharmaceuticals and Vertex.