450
Participants
Start Date
October 31, 2025
Primary Completion Date
June 30, 2032
Study Completion Date
June 30, 2032
migalastat HCl
Non-interventional study of participants receiving migalastat HCl 150 mg
ERT
Non-interventional study of participants receiving enzyme replacement therapy
Lysosomal and Rare Disorders Research and Treatment Center, Inc., Fairfax
Emory Genetics, Atlanta
Washington University School of Medicine, St Louis
Renal Disease Research Institute, Dallas
Lead Sponsor
Amicus Therapeutics
INDUSTRY