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Article History

Received: 12 September 2025

Accepted: 10 June 2026

First Online: 19 June 2026

Declarations

Ethics approval and consent to participate: The observational study was approved by an independent Institutional Review Board (IRB), Copernicus Group IRB, located in Research Triangle Park North Carolina, US (Protocol #20192014). The trial was performed in accordance with the provisions of the Declaration of Helsinki and Good Clinical Practice as outlined by the International Conference on Harmonization E6 (R2) and regional regulations. The clinical trial protocol was approved by the relevant ethics boards at each site. In the observational study, informed consent was obtained from all participants prior to participation in the web-based survey. Before enrollment in the phase 2 trial, written informed consent was obtained from the parent or legal guardian of each participant and written assent, if feasible given the participant’s age, from the participant.

Consent for publication: Not applicable.

Competing interests: M. Brod is a consultant to the pharmaceutical industry including Ascendis Pharma. L. McLeod is an employee of RTI Health Solutions (RTI International). A. Smith is an employee of Ascendis Pharma, Inc.