Supplemental Material, Manuscript_BrainInMPSSurvey_JIEMS_Appendix - Outcomes of a Physician Survey on the Type, Progression, Assessment, and Treatment of Neurological Disease in Mucopolysaccharidoses

<p>Supplemental Material, Manuscript_BrainInMPSSurvey_JIEMS_Appendix for Outcomes of a Physician Survey on the Type, Progression, Assessment, and Treatment of Neurological Disease in Mucopolysaccharidoses by Maurizio Scarpa, Paul R. Harmatz, Bianca Meesen, and Roberto Giugliani in Journal of Inborn Errors of Metabolism and Screening</p

Additional file 1: Table S1. of Clinical outcomes in idursulfase-treated patients with mucopolysaccharidosis type II: 3-year data from the hunter outcome survey (HOS)

Changes from baseline in clinical and biochemical parameters in patients with data available after 1, 2 or 3 years of treatment with idursulfase (DOCX 33 kb

Additional file 2: Table S2. of Clinical outcomes in idursulfase-treated patients with mucopolysaccharidosis type II: 3-year data from the hunter outcome survey (HOS)

Median urinary glycosaminoglycan (uGAG) levels and median changes from baseline in uGAG levels in patients with data available at consecutive timepoints (year 1, year 2 and year 3 of treatment with idursulfase) (DOCX 22 kb

g7-NPs/Alb representative confocal images of Ids-ko and wt mice brain.

<p>(a) Ids-ko and (b) syngeneic wt mice. Nuclei are shown in blue (DAPI staining), NPs in red (rhodamine labeling), Albumin in green (FITC labeling); last column represents the merged images.</p

Localization of g7-NPs/Alb in Idua-ko mouse brain.

<p>Representative confocal images of the g7-NPs/Alb perinuclear localization in the brain of Idua-ko mice injected with g7-NPs/Alb. (a) Albumin is shown in green (FITC labeling), (b) NPs in red (rhodamine labeling), (c) nuclei in blue (DAPI staining); (d) represents the merged images.</p

Chemico-physical characterization of nanoparticles.

<p>Chemico-physical characterization of nanoparticles.</p

Morphologic characterization of nanoparticles.

<p>AFM (panels a, c, e) and SEM (panels b, d, f) images of unloaded g7-NPs, used as control, loaded u-NPs/Alb and g7-NPs/Alb, respectively. g7-NPs: unloaded targeted nanoparticles; u-NPs/Alb: untargeted nanoparticles loaded with albumin;g7-NPs/Alb: targeted nanoparticles loaded with albumin.</p

Number of NPs in the brain of Idua-ko and wt mice.

<p>g7-NPs: unloaded targeted nanoparticles; u-NPs: unloaded untargeted nanoparticles; g7-NPs/Alb: targeted nanoparticles loaded with albumin: u-NPs/Alb, untargeted nanoparticles loaded with albumin; MIX1 (g7-NPs+Alb): unloaded targeted nanoparticles suspended in FITC-albumin solution; MIX2 (u-NPs+Alb): unloaded untargeted nanoparticles suspended in FITC-albumin solution. *p-value <0.05.</p

Number of g7-NPs/Alb in the brain of Ids-ko and wt mice.

<p>*p-value <0.001.</p

Type of injection performed in the two mouse models.

<p>Type of injection performed in the two mouse models.</p