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Authors
Keefe, Alexandra C. https://orcid.org/0009-0009-2455-1687
Jensen, Dana M.
Pham, Meranda M.
Au, Natalie Y T
Beckman, Erika
Penon-Portmann, Monica
Shelkowitz, Emily
Bend, Renee
Morrow, Michelle M.
Kruszka, Paul
Vats, Divya
Russell, Bianca E.
Chan, Erica
Wong, Derek
Rabani, Ahna
O’Grady, Lauren
Sahai, Inderneel
Widmeyer, Kimberly
Sperry, Ethan D.
Hallinan, Barbara E.
Tryon, Rebecca
Lund, Troy C.
Eichler, Florian S.
Sun, Angela
Bennett, James T.
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Article History
Received: 18 September 2024
Accepted: 22 April 2025
First Online: 9 May 2025
Competing interests
: Authors P.K. and M.M.M. are employees of GeneDx, LLC, but declare no financial or non-financial competing interests. A.R. is an employee at Natera but declares no financial or non-financial competing interests. F.S.E. is PI of an in vivo gene therapy trial in Canavan disease (Sponsor: ASPA Therapeutics), Co-PI of ex vivo lentiviral gene therapy trial in cerebral adrenoleukodystrophy (Sponsor: bluebird bio), Co-PI of PI of in vivo gene therapy trial in GM2 (Former Sponsor: Sio Therapeutics), Site-PI of Alexander Disease trial of ASOs (Sponsor: Ionis Pharmaceuticals), Consultant to Atlas Venture, Acadia Pharmaceuticals, Leal Therapeutics, Vigil, Takeda Therapeutics, Sanofi, SwanBio Therapeutics and UpToDate, founder of SwanBio Therapeutics, and is a NINDS Advisory Council Member but declares no financial or non-financial competing interests. The remaining authors declare no competing interests.
