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Authors
Nolen, Nicole
Aartsma-Rus, Annemieke
Caneva, Christine
Coughlin II, Curtis R.
Cousin, Margot A.
Douthwright, Catherine
Graessner, Holm
Kim-McManus, Olivia
Kuniholm, Ashley
Leonard, Stefanie
Martinsen, Andrea
Meserve, Margaret
Synofzik, Matthis
Yandava, Booma
Yu, Timothy W.
Demarest, Scott
Paxton, Roger J. https://orcid.org/0009-0003-5799-9492
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Funding
Funding for this research was provided by:
Chan Zuckerberg Initiative (DAF 2022-316467)
European Rare Disease Research Alliance (101156595)
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Article History
Received: 1 July 2025
Accepted: 29 April 2026
First Online: 21 May 2026
Declarations
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: Colorado Multiple Institutional Review Board 23-1605: Not human subject research.
: N/A.
: During the preparation of this work the author(s) used ChatGPT in order to improve readability. After using this tool/service, the author(s) reviewed and edited the content as needed and take(s) full responsibility for the content of the published article.
: Nicole Nolen is an employee of the N = 1 Collaborative, a nonprofit organization discussed in this manuscript. The N = 1 Collaborative is referenced as one of several examples of organizations supporting individualized therapy development. Several co-authors serve in unpaid advisory or volunteer roles within the N = 1 Collaborative. These affiliations are non-financial in nature. All authors declare that this affiliation did not influence the analysis nor conclusions presented.
