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Article History

Received: 1 May 2025

Accepted: 29 October 2025

First Online: 8 January 2026

Declarations

:

: Ethics approval was not needed for this study. Participants were invited to share comments that they were informed would be made publicly available as part of the EL-PFDD. Meeting pre-recordings, live-speaking during the EL-PFDD, and participation in the meeting (submission of comments and participation in polling questions) were entirely voluntary and implied consent.

: Attendance and involvement in the EL-PFDD was entirely voluntary, with attendees being informed that the outcome of the meeting would be made publicly available. As a result, consent for publication was not required.

: ASF, KM, FE, and RS have no conflicts of interest to disclose. AF, FEi, and KOSF are Co-Founders of ALD Connect. AF has received research funding as the site PI for clinical trials sponsored by Minoryx and Viking Therapeutics; is an institutional consultant for Vertex, Autobahn Therapeutics, Poxel Therapeutics, Sanofi, Spolia Therapeutics, Vigil Neuroscience, Takeda, SwanBio; and has served as a paid DSMB member for bluebird bio (now Genetix Biotherapeutics). FEi was a PI for ASPA Therapeutics; a co-PI for bluebird bio, Sio Therapeutics and UMASS Chan Medical School; a site PI for Ionis Pharmaceuticals and the n-Lorem Foundation; a consultant to Atlas Venture, Acadia Pharmaceuticals, Leal Therapeutics, Orchard Therapeutics, Sanofi Pharmaceuticals, Spolia Therapeutics, Spur Therapeutics, Takeda Therapeutics, UpToDate, and Vigil Neuro; and the Founder of SwanBio Therapeutics (now Spur Therapeutics).