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Original Research

An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naïve to enzyme replacement therapy

, , , , , , & show all
Pages 1771-1781 | Published online: 25 May 2016

Figures & data

Table 1 Demographic and baseline clinical characteristics

Table 2 Summary of efficacy results for the only patient who tested positive for both IgG and neutralizing antibodies

Figure 1 Heart rate variability as assessed by SDNN: individual values at baseline and at 55 weeks.

Notes: (A) Observed values of SDNN (ms). (B) SDNN Z-scores. SDNN represents the standard deviation of all filtered RR intervals for the length of the analysis. The different colored shapes within the figure show individual patient data at baseline and 55 weeks.
Figure 1 Heart rate variability as assessed by SDNN: individual values at baseline and at 55 weeks.

Figure 2 LVMI over time for the patient who had LVH at baseline.

Note: SDNN represents the standard deviation of all filtered RR intervals for the length of the analysis.
Abbreviations: LVH, left ventricular hypertrophy; LVMI, left ventricular mass index.
Figure 2 LVMI over time for the patient who had LVH at baseline.

Figure 3 Correlation between change from baseline to week 55 in SDNN and LVMI in patients with baseline SDNN <100 ms.

Notes: *r = Pearson correlation coefficient. SDNN represents the standard deviation of all filtered RR intervals for the length of the analysis.
Abbreviation: LVMI, left ventricular mass index.
Figure 3 Correlation between change from baseline to week 55 in SDNN and LVMI in patients with baseline SDNN <100 ms.

Figure 4 Mean (SE) change from baseline in eGFR.

Note: The safety population comprises all enrolled patients who received at least one dose of agalsidase alfa.
Abbreviations: eGFR, estimated glomerular filtration rate; SE, standard error; SP, safety population.
Figure 4 Mean (SE) change from baseline in eGFR.

Figure 5 Mean plasma Gb3 over time for the overall population and by sex.

Notes: (A) Observed values (nmol/mL). (B) Change from baseline.
Abbreviation: Gb3, globotriaosylceramide.
Figure 5 Mean plasma Gb3 over time for the overall population and by sex.