• Issue

    Muscle & Nerve: Volume 72, Issue 2

    179-345
    August 2025

ISSUE INFORMATION

CLINICAL RESEARCH ARTICLE

Open Access

Switching Enzyme Replacement Therapy for Late-Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of PROPEL

  • Pages: 230-239
  • First Published: 07 May 2025
Switching Enzyme Replacement Therapy for Late-Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of PROPEL

A total of 95 ERT-experienced adults with LOPD were randomized to switch to cipaglucosidase alfa + miglustat or remain on alglucosidase alfa treatment. After 52 weeks, patients remaining on alglucosidase alfa showed worsening or stability for most outcomes, whereas patients who switched to cipaglucosidase alfa + miglustat generally showed stability or improvement.

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  • Page: 345
  • First Published: 26 July 2025