Clinical characteristics and somatic burden of patients with mucopolysaccharidosis II with or without neurological involvement: An analysis from the Hunter Outcome Survey.

"Declaration of Competing Interest Heather Lau has received consulting fees from Amicus Therapeutics, Pfizer, Sanofi Genzyme, Takeda, and Taysha and research grants from Amicus Therapeutics, BioMarin, Pfizer, Protalix Biotherapeutics, Sangamo Therapeutics, Sanofi Genzyme, Takeda, and Ultragenyx, and is a full-time employee and stockholder of Ultragenyx Pharmaceutical. Paul Harmatz has received honoraria, consulting fees, and/or research grants from Adrenas Aeglea, Allievex, Amicus Therapeutics, Ascendis Pharma, Aspa therapeutics, Astellas Gene Therapies, AVROBIO, Azafaros, BioMarin, Calcilytix, Capsida Biotherapeutics, Chiesi, Denali Therapeutics, EdiGene, Grace Science, Homology Medicines, Inventiva Pharma, JCR Pharmaceuticals, Novel Pharma, Orchard Therapeutics, Orphazyme, Paradigm, QED Therapeutics, RallyBio, REGENXBIO, Renoviron, Sangamo Therapeutics, SalioGen Therapeutics, Sanofi Genzyme, Sobi and Takeda. Bianca Link has received fees and travel grants from Alexion, Sanofi Genzyme, Shire, and Takeda. Jaco Botha is a full-time employee of Takeda Pharmaceuticals International AG and stockholder of Takeda Pharmaceutical Company Limited. Jennifer Audi was a full-time employee of Takeda Pharmaceuticals International AG (now with Ultragenyx Europe GmbH) and was a stockholder of Takeda Pharmaceutical Company Limited."

"Funding This study was funded by Takeda Pharmaceuticals International AG. Collection and analysis of data in HOS is supported by Takeda Pharmaceuticals International AG and data analyses were performed by Takeda Pharmaceuticals International AG under the direction of the authors. Medical writing assistance, provided under the direction of the authors, was funded by Takeda Development Center Americas, Inc."