Effect Size Analysis of Cipaglucosidase Alfa Plus Miglustat Versus Alglucosidase Alfa in ERT-experienced Adults with Late-onset Pompe Disease in PROPEL (S21.003).

miglustat - Generic mfg.

Pombiliti (cipaglucosidase alfa-atga) - Amicus

Myozyme (alglucosidase alfa) - Sanofi

Cabaletta (trehalose) - Seelos Therap

http://www.ncbi.nlm.nih.gov/pubmed/39977945

Antonio Toscano; Barry Byrne; Benedikt Schoser; Drago Bratkovic; Frederick Holdbrook; Hani Kushlaf; Jeff Castelli; Jordi Díaz-Manera; Kristl Claeys; Mark Roberts; Mazen Dimachkie; Priya Kishnani; Sheela Das; Tahseen Mozaffar; Yasmine Wasfi

Feb 20, 2025