About the Authors

Renuka Pudi Limgala

rlimgala@ldrtc.org

Affiliations Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, Virginia, United States of America, Amerimmune, O and O Alpan, LLC, Fairfax, Virginia, United States of America

Chidima Ioanou

Affiliation Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, Virginia, United States of America

Matthew Plassmeyer

Affiliation Amerimmune, O and O Alpan, LLC, Fairfax, Virginia, United States of America

Mark Ryherd

Affiliation Amerimmune, O and O Alpan, LLC, Fairfax, Virginia, United States of America

Lina Kozhaya

Affiliation Jackson Laboratory for Genomic Medicine, Farmington, Connecticut, United States of America

Lauren Austin

Affiliation Amerimmune, O and O Alpan, LLC, Fairfax, Virginia, United States of America

Cem Abidoglu

Affiliation Jackson Laboratory for Genomic Medicine, Farmington, Connecticut, United States of America

Derya Unutmaz

Affiliation Jackson Laboratory for Genomic Medicine, Farmington, Connecticut, United States of America

Oral Alpan

Affiliation Amerimmune, O and O Alpan, LLC, Fairfax, Virginia, United States of America

Ozlem Goker-Alpan

Affiliation Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, Virginia, United States of America

Competing Interests

Financial support from Amerimmune, O & O Alpan, LLC in the form of salaries and Sanofi-Genzyme in the form of grant funding do not alter our adherence to all PLOS ONE policies on sharing data and materials.

Author Contributions

Conceptualization: OGA. Formal analysis: RPL LK CA. Funding acquisition: OGA. Investigation: RPL CI MR LK LA CA OGA. Methodology: RPL MP DU OA. Project administration: RPL CI OGA. Resources: MP DU OA OGA. Supervision: RPL DU OGA. Writing – original draft: RPL OGA. Writing – review & editing: DU OA.