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migalastat

National Institutes of Health

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6 relations

Broader (1)

1-Deoxynojirimycin
1-deoxy-galactonojirimycinN-butyldeoxygalacto-nojirimycinanalogs & derivativesmigalastat hydrochloride

Narrower (1)

GR181413A

Papers overview

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2020
2020
Treatment of Fabry's Disease With Migalastat: Outcome From a Prospective Observational Multicenter Study (FAMOUS)
Fabry's disease (FD) is an X‐linked lysosomal storage disorder caused by the deficient activity of the lysosomal enzyme… 
Review
2020
Review
2020
The GALA project: practical recommendations for the use of migalastat in clinical practice on the basis of a structured survey among Italian experts
Background Oral migalastat has recently been approved for the treatment of Anderson-Fabry disease (FD) in patients aged ≥16 years… 
2019
2019
Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study
Background Fabry disease is a progressive X-linked lysosomal disorder. In this subgroup analysis of the global phase III ATTRACT… 
2018
2018
Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial
BackgroundFabry disease is frequently characterized by gastrointestinal symptoms, including diarrhea. Migalastat is an orally… 
2018
2018
Correction: Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study
2017
2017
Reduction of podocyte globotriaosylceramide content in adult male patients with Fabry disease with amenable GLA mutations following 6 months of migalastat treatment
Objective Deficiency of α-galactosidase A (αGal-A) in Fabry disease leads to the accumulation mainly of globotriaosylceramide… 
Highly Cited
2016
Highly Cited
2016
Migalastat: First Global Approval
Migalastat (Galafold™)—a small molecule drug developed by Amicus Therapeutics that restores the activity of specific mutant forms… 
2016
2016
Fabry disease: A pharmacological chaperone on the horizon
For more than a decade, enzyme replacement therapy represented the only treatment option for patients with Fabry disease. New… 
2015
2015
Relative bioavailability and the effect of meal type and timing on the pharmacokinetics of migalastat in healthy volunteers
Migalastat HCl is an investigational, pharmacological chaperone for mutant α‐galactosidase A, which is responsible for Fabry… 
2013
2013
Pharmacokinetics and Safety of Migalastat HCl and Effects on Agalsidase Activity in Healthy Volunteers
Migalastat HCl is an investigational, oral treatment for Fabry disease, an X‐linked lysosomal storage disorder. Four Phase 1… 
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