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The funder was involved in the study design, data collection, data analysis, data interpretation, and writing of all related reports and publications.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Funding"}},{"value":"John Day, Jerry Mendell, Eugenio Mercuri, Richard Finkel, and Kevin A. Strauss were study investigators in onasemnogene abeparvovec clinical trials. Aaron Kleyn, Sitra\u00a0Tauscher-Wisniewski, Francis Fonyuy Tukov, Sandra P. Reyna, and Deepa H. Chand are employees of Novartis Gene Therapies, Inc., the manufacturer of onasemnogene abeparvovec.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflicts of interest"}},{"value":"Novartis is committed to sharing clinical trial data with external researchers and has been doing so voluntarily since 2014. Novartis was the third member to join ClinicalStudyDataRequest.com (CSDR), which is the first data-sharing consortium of clinical study sponsors and funders. CSDR is a leader in the data-sharing community inspired to drive scientific innovation and improve medical care by facilitating access to patient-level data from clinical studies (). Novartis is committed to sharing, upon requests from qualified external researchers and subsequent approval by an independent review panel based upon scientific merit, anonymized patient-level and study-level clinical trial data, and redacted clinical study reports, for medicines and indications approved in the United States and Europe after the respective study is accepted for publication. All data provided are anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described at.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Availability of data and material"}},{"value":"Not Applicable.","order":5,"name":"Ethics","group":{"name":"EthicsHeading","label":"Code Availability"}},{"value":"DHC and FFT, both of Novartis Gene Therapies, Inc., developed the first draft of the manuscript and conducted the data analysis. All authors reviewed the manuscript for important intellectual content and approved the final version for submission. DHC had full access to all the data in the study and takes responsibility for the integrity of the data and the accuracy of the data analysis. All authors had full access to all the data in the study and accept responsibility to submit for publication. All authors reviewed and approved the final version of the manuscript.","order":6,"name":"Ethics","group":{"name":"EthicsHeading","label":"Author contributions"}},{"value":"All study protocols were approved by the institutional review boards or ethics committees, with appropriate informed consent obtained. Studies were registered with(NCT02122952 [START], NCT03306277 [STR1VE-US], NCT03505099 [SPR1NT], NCT03461289 [STR1VE-EU], NCT03837184 [STR1VE-AP], NCT03421977 [LT-001], and NCT04042025 [LT-002]).","order":7,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval"}},{"value":"Not applicable.","order":8,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent to participate"}},{"value":"All authors have consented to submission for publication. The decision to publish was theirs alone.","order":9,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}}]}}