Challenges in multinational rare disease clinical studies during COVID-19: regulatory assessment of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease.

miglustat - Generic mfg.

Pombiliti (cipaglucosidase alfa-atga) - Amicus

Myozyme (alglucosidase alfa) - Sanofi

http://www.ncbi.nlm.nih.gov/pubmed/39775064

; Benedikt Schoser; Fred Holdbrook; Jordi Díaz-Manera; Mitchell Goldman; Ryan Graham; Shahram Attarian

Jan 12, 2025