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July 17, 2026Journal of Comparative Effectiveness ResearchOpen Access

A disease progression model comparing the long-term mobility and respiratory outcomes of adults with late-onset Pompe disease receiving cipaglucosidase alfa plus miglustat versus alglucosidase alfa

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Authors

ADAmy DymondWGWill GreenMCMatthew Crabtree

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Overview

Randomized trial compares long-term mobility and respiratory outcomes in adults with LOPD, suggesting cipaglucosidase alfa plus miglustat may offer benefits over alglucosidase alfa.

Key Points

  • The aim is to compare long-term mobility and respiratory outcomes of different treatments in adults with late-onset Pompe disease.
  • Developed a patient-level simulation model to predict lifetime outcomes based on 6-min walk distance and forced vital capacity.
  • Used data from clinical studies NCT03729362 and NCT02675465 for outcomes over four years for cipaglucosidase alfa plus miglustat and one year for alglucosidase alfa.
  • Informed by French Pompe disease registry data for long-term predictions.
  • Cipaglucosidase alfa plus miglustat is predicted to provide an additional 2.72 years without mobility or respiratory support compared to alglucosidase alfa.
  • Patients receiving alglucosidase alfa may require wheelchair dependency for an additional 2.57 years and invasive respiratory support for 1.55 years.

Cite This Study

Dymond et al. (2026) studied this question.

synapsesocial.com/papers/6a59c664a58755010b4718e5https://doi.org/10.57264/cer-2025-0202
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Also Consider

Synapse has enriched 3 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07)2024 · 30 citations
  2. 2Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational study2013 · 124 citations
  3. 3Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02)2023 · 22 citations