MigalastatFabry diseaseUniversity Hospital Birmingham
…ceramide (GB3), causing progressive, multisystem tissue damage. Migalastat is an oral chaperone therapy that stabilises defective enzyme i…
3articles mentioning drugs
16drug mentions
1distinct drugs extracted
0%of mentions human-reviewed
Active filtersEntity: Migalastat ×
Migalastat
This drug is selected. Its supporting articles are shown below.
Articles mentioning Migalastat
3 articles · page 1 of 198.3%
migalastatFabry disease (FD)
…Gal A enzyme activity was significantly reduced. Treatment with migalastat, a pharmacological chaperone for α-Gal A, partially restored th…
migalastatFabry diseaseATTRACTNCT00925301
…sease severity and burden and support the long-term efficacy of migalastat for the treatment of females.…