Vázquez-Costa, Juan F.
Branas-Pampillón, María
Medina-Cantillo, Julita
Povedano, Mónica
Pitarch-Castellano, Inmaculada
López-Lobato, Mercedes
Fernández-Ramos, Joaquín A.
Lafuente-Hidalgo, Miguel
Rojas-García, Ricard
Caballero-Caballero, José M.
Málaga, Ignacio
Eirís-Puñal, Jesús
De Lemus, Mencía
Cattinari, María G.
Cabello-Moruno, Rosana
Díaz-Abós, Paola
Sánchez-Menéndez, Victoria
Rebollo, Pablo
Maurino, Jorge
Madruga-Garrido, Marcos
Funding for this research was provided by:
Roche España
Article History
Received: 5 September 2022
Accepted: 4 October 2022
First Online: 21 October 2022
Declarations
:
: The authors would like to thank the parents and children with SMA whose support and collaboration made the SMA-TOOL study possible.
: This study was funded by the Medical Department of Roche Farma, Spain. The sponsor also funded the journal’s Rapid Service.
: All authors made a significant contribution to the work reported in study conception, design, execution, acquisition of data, and analysis and interpretation. All contributed to the article’s drafting, revision, or critical review; all gave final approval of the version to be published; all agreed on the journal to which the article was submitted; and all agreed to be held accountable for all aspects of the work.
: María Branas-Pampillón, Rosana Cabello-Moruno, Paola Díaz-Abós, Victoria Sánchez-Menéndez, and Jorge Maurino are employees of Roche Farma, Spain. Pablo Rebollo is an employee of IQVIA Spain. Juan F Vázquez-Costa, Julita Medina-Cantillo, Mónica Povedano, Inmaculada Pitarch-Castellano, Mercedes López-Lobato, Joaquín A Fernández-Ramos, Miguel Lafuente-Hidalgo, Ricard Rojas-García, José M Caballero-Caballero, Ignacio Málaga, Jesús Eirís-Puñal, Mencía De Lemus, María G Cattinari, and Marco Madruga-Garrido declare no conflict of interest.
: The SMA-TOOL study was conducted in accordance with the Good Clinical Practice Guidelines of the International Conference on Harmonisation and with the ethical principles of the Declaration of Helsinki and was approved by the investigational review board of the Hospital Universitari de Bellvitge (Barcelona, Spain; reference code: PR264/20). Written informed consent was obtained from all subjects.
: Qualified researchers may request access to individual patient-level data through the corresponding author. The datasets generated during the analysis of the study are available from the corresponding author on reasonable request.