| Pompe disease places a substantial economic burden on patients, families, healthcare systems, and society. |
| The majority of costs associated with the management of Pompe disease are driven by enzyme-replacement therapy (ERT), the only currently approved treatment. |
| The incremental cost-effectiveness ratio (incremental cost per quality-adjusted life-year gained) of ERT ranges widely, but is consistently high when compared with established cost-effectiveness thresholds. |
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