Add to ORKGMigalastat HCl (AT1001, 1-Deoxygalactonojirimycin) is an investigational pharmacological chaperone for the treatment of α-galactosidase A (α-Gal A) deficiency, which leads to Fabry disease, an X-linked, lysosomal storage disorder. The currently approved, biologics-based therapy for Fabry disease is enzyme replacement therapy (ERT) with either agalsi-dase alfa (Replagal) or agalsidase beta (Fabrazyme). Based on preclinical data, migalastat HCl in combination with agalsidase is expected to result in the pharmacokinetic (PK) enhancement of agalsidase in plasma by increasing the systemic exposure of active agalsi-dase, thereby leading to increased cellular levels in disease-relevant tissues. This Phase 2a study design consisted of an open-label...
Fabry disease (FD) results from mutations in the gene ( GLA ) that encodes the lysosomal enzyme α-ga...
BACKGROUND Fabry's disease, an X-linked disorder of lysosomal alpha-galactosidase deficiency, leads ...
PURPOSE: Fabry disease is an X-linked lysosomal storage disorder caused by mutations in the α-galact...
<div><p>Migalastat HCl (AT1001, 1-Deoxygalactonojirimycin) is an investigational pharmacological cha...
UNLABELLED: Migalastat HCl (AT1001, 1-Deoxygalactonojirimycin) is an investigational pharmacological...
BACKGROUND: Fabry disease (FD) is a genetic disorder resulting from deficiency of the lysosomal enzy...
BACKGROUND: Fabry disease is an X-linked lysosomal storage disorder caused by GLA mutations, resulti...
Abstract Background Fabry disease (FD) is a genetic disorder resulting from deficiency of the lysoso...
International audienceBACKGROUND: Fabry disease (OMIM 301500) is an X-linked disorder caused by alph...
Fabry disease is a lysosomal storage disorder caused by the deficiency of the α-galactosidase-A enzy...
: Fabry disease (FD) is an X-linked lysosomal storage disorder resulting from the deficiency of the ...
Fabry's disease (FD) is an X-linked lysosomal storage disorder caused by the deficient activity of t...
Fabry disease (FD) results from mutations in the gene (GLA) that encodes the lysosomal enzyme a-gala...
Fabry disease (FD) results from mutations in the gene (GLA) that encodes the lysosomal enzyme α-gala...
Fabry disease (FD) results from mutations in the gene (GLA) that encodes the lysosomal enzyme a-gala...
Fabry disease (FD) results from mutations in the gene ( GLA ) that encodes the lysosomal enzyme α-ga...
BACKGROUND Fabry's disease, an X-linked disorder of lysosomal alpha-galactosidase deficiency, leads ...
PURPOSE: Fabry disease is an X-linked lysosomal storage disorder caused by mutations in the α-galact...
<div><p>Migalastat HCl (AT1001, 1-Deoxygalactonojirimycin) is an investigational pharmacological cha...
UNLABELLED: Migalastat HCl (AT1001, 1-Deoxygalactonojirimycin) is an investigational pharmacological...
BACKGROUND: Fabry disease (FD) is a genetic disorder resulting from deficiency of the lysosomal enzy...
BACKGROUND: Fabry disease is an X-linked lysosomal storage disorder caused by GLA mutations, resulti...
Abstract Background Fabry disease (FD) is a genetic disorder resulting from deficiency of the lysoso...
International audienceBACKGROUND: Fabry disease (OMIM 301500) is an X-linked disorder caused by alph...
Fabry disease is a lysosomal storage disorder caused by the deficiency of the α-galactosidase-A enzy...
: Fabry disease (FD) is an X-linked lysosomal storage disorder resulting from the deficiency of the ...
Fabry's disease (FD) is an X-linked lysosomal storage disorder caused by the deficient activity of t...
Fabry disease (FD) results from mutations in the gene (GLA) that encodes the lysosomal enzyme a-gala...
Fabry disease (FD) results from mutations in the gene (GLA) that encodes the lysosomal enzyme α-gala...
Fabry disease (FD) results from mutations in the gene (GLA) that encodes the lysosomal enzyme a-gala...
Fabry disease (FD) results from mutations in the gene ( GLA ) that encodes the lysosomal enzyme α-ga...
BACKGROUND Fabry's disease, an X-linked disorder of lysosomal alpha-galactosidase deficiency, leads ...
PURPOSE: Fabry disease is an X-linked lysosomal storage disorder caused by mutations in the α-galact...