Study concept and design: All authors.
Acquisition, analysis, or interpretation of data: All authors.
Drafting of the manuscript: All authors.
Critical revision of the manuscript for important intellectual content: All authors.
Administrative, technical, or material support: All authors.
Study supervision: Pascual, Park.
Conflict of Interest Disclosures: Dr Park is a member of the Scientific Advisory Board of Fujirebio Inc. No other disclosures were reported.
Funding/Support: Drs Pascual and Park are supported by National Institutes of Health Office of Rare Diseases Research: Collaboration, Education, and Test Translation (CETT) program for rare genetic diseases.
Role of the Sponsor: The National Institutes of Health had no role in the design and conduct of the study; collection, management, analysis, and interpretation of the data; preparation, review, or approval of the manuscript; and decision to submit the manuscript for publication.