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February 28, 2026Therapeutic Advances in Rare DiseaseOpen Access

Miglustat: a first-in-class enzyme stabilizer for cipaglucosidase alfa for the treatment of late-onset Pompe disease

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Authors

HDHolly DubbsBBBarry J. ByrneSASampath Arepalli

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Overview

Clinical data reveals improved treatment outcomes in late-onset Pompe disease with miglustat and cipaglucosidase alfa, suggesting enhanced therapy efficacy.

Key Points

  • The study aims to evaluate the efficacy and safety of miglustat in combination with cipaglucosidase alfa for late-onset Pompe disease.
  • Utilized preclinical studies in Pompe mouse models
  • Conducted clinical trials to assess safety and efficacy in patients with Pompe disease
  • Examined functional outcomes and biomarker levels in patients using the combination therapy
  • Cipaglucosidase alfa exposure improved with miglustat co-administration
  • Enhanced uptake into target tissues was observed
  • Functional outcomes showed significant improvement compared to cipaglucosidase alfa alone
  • Miglustat was well tolerated with fewer gastrointestinal events when dosed bi-weekly

Cite This Study

Dubbs et al. (2026) studied this question.

synapsesocial.com/papers/69a287570a974eb0d3c0306bhttps://doi.org/10.1177/26330040261425686
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease: results from multiple clinical trials2026
  2. 2A 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 alfa2026
  3. 3Cipaglucosidase alfa-atga: Unveiling new horizons in Pompe disease therapy2024 · 2 citations
  4. 4104-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 · 29 citations
  5. 5Comparing the efficacy of cipaglucosidase alfa plus miglustat with alglucosidase alfa for late-onset Pompe disease: an expanded network meta-analysis utilizing patient-level and aggregate data2026