Bibliographical noteFunding Information:
Eisengart: Research support from Lysogene, Sangamo and Shire/Takeda; consultant to Denali Therapeutics, Sangamo, Sanofi Genzyme, and Shire/Takeda; advisory boards for Amicus, bluebird bio, Orchard Therapeutics, and Sanofi Genzyme; contract work for Shapiro Neuropsychology Consulting, LLC.
King: Research support from Alexion Pharmaceuticals, Inc., Shire/Takeda, and Sanofi Genzyme, and contract work for Shapiro Neuropsychology Consulting, LLC.
This investigator-initiated research was supported by a grant from Shire Human Genetic Therapies Inc. , Lexington, MA, a member of the Takeda group of companies through # IIR-USA-001644 , and by NIH U54NS065768 . Historic chart review and analysis was funded by NIH U54NS065768 . The Lysosomal Disease Network (U54NS065768) is a part of the Rare Diseases Clinical Research Network, an initiative of the Office of Rare Diseases Research, and the National Center for Advancing Translational Sciences. This consortium is funded through a collaboration between the National Center for Advancing Translational Sciences , the National Institute of Neurological Disorders and Stroke , and the National Institute of Diabetes and Digestive and Kidney Diseases . Data curation was supported by the National Institutes of Health's National Center for Advancing Translational Sciences , grant UL1TR002494 . The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health's National Center for Advancing Translational Sciences. Resources for convening the caregiver advisory board and sharing of data was provided by Denali Therapeutics (South San Francisco, CA). The advisory board was facilitated by the first author (JBE). Infrastructure support was provided by the National MPS Society, Project Alive, MPS Superhero Foundation, the University of Minnesota Department of Pediatrics, and the University of Minnesota's Center for Neurobehavioral Development. None of the funding sources had any role in study design; collection, analysis and interpretation of data; writing or revision of the original manuscript; the decision to submit the article for publication; nor writing or revision of the Response to Mr. Grant.
- Behavioral decline
- Hunter syndrome
- Mucopolysaccharidosis II
- Neurobehavioral symptoms
- Neuronopathic phenotype
PubMed: MeSH publication types
- Journal Article