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Authors
Rahm, Lisa https://orcid.org/0000-0003-1420-9027
Hale, Melissa A.
Raaijmakers, Renée H. L. https://orcid.org/0000-0001-9495-4830
Marrero Quiñones, Alexandra
Patki, Tejal
Johnson, Nicholas E.
van Bokhoven, Hans
Mul, Karlien https://orcid.org/0000-0001-8487-9478
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Article History
Accepted: 1 September 2025
First Online: 22 September 2025
Competing interests
: K.M. served as a paid consultant for Dyne Therapeutics and Avidity Biosciences (payments to institution). She receives research funding from PepGen Inc. N.E.J. has received grant funding from NINDS (R01NS104010, U01NS124974), NCATS (R21TR003184), the CDC (U01DD001242) and the FDA (7R01FD006071). N.E.J. receives royalties from the CCMDHI and the CMTHI. He receives research funds from Novartis, Takeda, PepGen, Sanofi Genzyme, Dyne, Vertex Pharmaceuticals, Fulcrum Therapeutics, AskBio, ML Bio, and Sarepta. N.E.J. has consulted for Arthex, Angle Therapeutics, Juvena, Rgenta, PepGen, AMO Pharma, Takeda, Design, Dyne, AskBio, Avidity and Vertex Pharmaceuticals. He has equity in Repeat RNA Therapuetics, Evolyra Therapeutics, and Juvena Therapeutics. M.A.H. has consulted for Juvena Therapeutics, Arrakis Therapeutics and Vertex Pharmaceuticals and retains equity in Evolyra Therapeutics. The other authors declare no competing interests.
