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. 2014 Aug 13;1:14033. doi: 10.1038/mtm.2014.33

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Copyright © 2014 American Society of Gene & Cell Therapy

This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivs 3.0 Unported License. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit http://creativecommons.org/licenses/by-nc-nd/3.0/

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Timeline: At 5.5 months of age the patient received an induction dose of Rituximab followed by daily oral Sirolimus. A monthly dose of IV immunoglobulin (IVIG) was also given. The patient continued the B-cell depletion regimen with Rituximab at 12 weeks intervals while receiving Sirolimus daily. At 6 months of age, following completion of Rituximab, the patient started on enzyme replacement therapy (ERT) at a dose of 20 mg/kg every 7–10 days. A brief course of methylprednisolone was given prior to and following AAV1-GAA intramuscular gene transfer to the diaphragm at 45 months of age.