Fig. 1: Kaplan–Meier survival analysis of cross-reactive immunological material (CRIM)-negative infantile Pompe disease (IPD) patients treated with enzyme replacement therapy (ERT) monotherapy compared with those treated ERT + immune tolerance induction (ITI) (combined and by individual groups). | Genetics in Medicine

Fig. 1: Kaplan–Meier survival analysis of cross-reactive immunological material (CRIM)-negative infantile Pompe disease (IPD) patients treated with enzyme replacement therapy (ERT) monotherapy compared with those treated ERT + immune tolerance induction (ITI) (combined and by individual groups).

From: Transforming the clinical outcome in CRIM-negative infantile Pompe disease identified via newborn screening: the benefits of early treatment with enzyme replacement therapy and immune tolerance induction

Fig. 1

(a,b) Comparison of overall and invasive ventilator–free survival in all patients treated with ERT + ITI (n = 20) versus those treated with ERT monotherapy (n = 11). Both overall and invasive ventilator–free survival were significantly prolonged in patients treated with ERT + ITI (p < 0.0001). (c,d) Comparison of overall and invasive ventilator–free survival in early (n = 5), intermediate (n = 7), and late (n = 8) treatment groups, and the ERT monotherapy group (n = 11).

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