Nagy, Sara http://orcid.org/0000-0002-3508-860X
Hafner, Patricia
Schmidt, Simone
Rubino-Nacht, Daniela
Schädelin, Sabine
Bieri, Oliver
Fischer, Dirk
Funding for this research was provided by:
Swiss National Science Foundation
Scientific and Technological Research Council of Turkey
French National Research Agency, ERA-Net for Research on Rare Diseases
Duchenne UK
Duchenne Parent Project Netherlands
Association Monégasque contre les Myopathies
Article History
Received: 17 May 2019
Accepted: 21 September 2019
First Online: 21 November 2019
Ethics approval and consent to participate
: The trial was approved by the local ethics committee (ethics committee of both Basel cantons, 2017–01708), the National Swiss Drug Agency (Swissmedic, 2018DR3068), the European Union Drug Regulating Authorities Clinical Trials (EudraCT 2017–004554-42), and the European Union Drug Regulating Authorities Pharmacovigilance (EudraVigilance). The study was registered at ClinicalTrials.gov (NCT03354039) and at the Swiss National Clinical Trials Portal (SNCTP000002387) prior to recruitment. Patients will be informed about preclinical data, risks and possible benefits of the study. The participation in this study is voluntary. Written informed consent from patients and/or their caregivers will be obtained.
: Not applicable.
: DF is the principle investigator of studies on spinal muscular atrophy sponsored by Hofmann-La Roche Ltd. There are no other activities related to commercial companies. The remaining authors declare that they have no competing interests.