migalastat

a potent inhibitor of glycolipid biosynthesis

Also Known As:

1-butyl-2-(hydroxylmethyl)piperidine-3,4,5-triol; 1-deoxy-galactonojirimycin; 1-deoxygalacto-nojirimycin; 1-deoxygalactonojirimycin; AT1001 deoxyjirimycin; GR181413A; Galafold; N-butyldeoxygalacto-nojirimycin; N-butyldeoxygalactonojirimycin; NB-DGJ; lucerastat; migalastat HCl; migalastat hydrochloride

Networked: 102 relevant articles (9 outcomes, 18 trials/studies)

Relationship Network

Bio-Agent Context: Research Results

Experts

1.	Schiffmann, Raphael: 14 articles (12/2021 - 07/2007)
2.	Bichet, Daniel G: 12 articles (12/2021 - 01/2015)
3.	Skuban, Nina: 12 articles (12/2021 - 08/2016)
4.	Feldt-Rasmussen, Ulla: 8 articles (12/2021 - 08/2016)
5.	Brand, Eva: 7 articles (01/2022 - 01/2019)
6.	Lenders, Malte: 7 articles (01/2022 - 01/2019)
7.	Giugliani, Roberto: 7 articles (12/2021 - 08/2016)
8.	Barth, Jay A: 7 articles (09/2020 - 11/2017)
9.	Ishii, Satoshi: 7 articles (01/2012 - 11/2004)
10.	Nordbeck, Peter: 6 articles (01/2022 - 01/2018)

Related Diseases

1.	Fabry Disease (Fabry's Disease) 01/01/2009 - "One of the most potent inhibitors of alpha-gal A, 1-deoxygalactonojirimycin, has also been shown to be effective in enhancing residual alpha-gal A activity in cultured fibroblasts and lymphoblasts established from patients with Fabry disease caused by a variety of missense mutations. " 01/01/2018 - "A marked decrease in plasma GSLs was observed, suggesting clinical potential for lucerastat in patients with Fabry disease." 01/01/2023 - "A small but significant reduction in left ventricular (LV) mass after 18 months of migalastat treatment has been reported in Fabry disease (FD). " 01/01/2019 - "Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable variants: data from the phase 3 randomized, multicenter, double-blind clinical trial and extension study." 09/01/2019 - "The migalastat GLP-HEK assay is the gold standard for determining amenability in patients with Fabry disease."
2.	Sandhoff Disease (Sandhoff's Disease) 08/01/2004 - "Improved outcome of N-butyldeoxygalactonojirimycin-mediated substrate reduction therapy in a mouse model of Sandhoff disease." 05/01/2008 - "NB-DGJ treatment, administered from postnatal day 2 (p-2) to p-5 (600 mg/kg/day)), significantly reduced total brain ganglioside and GM2 content in the Sandhoff disease (Hexb(-/-)) mice, but did not reduce the content of GA2. " 05/01/2008 - "N-butyldeoxygalactonojirimycin reduces brain ganglioside and GM2 content in neonatal Sandhoff disease mice." 08/01/2004 - "In the present study, a more selective compound, the galactose analogue N-butyldeoxygalactonojirimycin (NB-DGJ), was evaluated in the Sandhoff disease mouse model. "
3.	Diarrhea 01/01/2018 - "After 6 months, significantly more patients receiving migalastat versus placebo experienced improvement in diarrhea based on a MCID of 0.33 (43% vs 11%; p = .02), including the subset with baseline diarrhea (71% vs 20%; p = .02). " 01/01/2018 - "Migalastat was associated with a clinically meaningful improvement in diarrhea in patients with Fabry disease and amenable mutations. " 01/01/2018 - "We evaluated minimal clinically important differences (MCID) in diarrhea based on the corresponding domain of the patient-reported Gastrointestinal Symptom Rating Scale (GSRS) in patients with Fabry disease and amenable mutations (N = 50) treated with migalastat 150 mg every other day or placebo during the phase 3 FACETS trial (NCT00925301). " 01/01/2018 - "Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial."
4.	Cardiovascular Diseases (Cardiovascular Disease) 08/28/2023 - "Disease-specific therapy, including enzyme replacement therapy (ERT) and chaperone therapy (migalastat), is recommended for patients exhibiting cardiovascular involvement, but its efficacy for modulating cardiovascular disease expression and optimal timing of initiation remains to be fully established. "
5.	Gaucher Disease (Gaucher's Disease) 10/28/1994 - "In an in vitro Gaucher's disease model NB-DGJ was as effective as NB-DNJ in preventing glycolipid storage and may represent a more selective potential therapeutic agent than NB-DNJ for the management of this and other glycosphingolipidoses." 04/01/2023 - "In this study, we screened the anti-FMD effects of various iminosugars and showed that some compounds, including N-butyldeoxynojirimycin (NB-DNJ, miglustat, an inhibitor of glucosylceramide synthase (GCS)), a clinically approved drug for treating Niemann-Pick disease type C and Gaucher disease type 1, inhibited TGF-β1-induced FMD by inhibiting the nuclear translocation of Smad2/3. N-butyldeoxygalactonojirimycin having GCS inhibitory effect did not attenuate the TGF-β1-induced FMD, suggesting that NB-DNJ exerts the anti-FMD effects by GCS inhibitory effect independent manner. " 11/01/2008 - "Since both evidence support a link between CFTR function and inflammation, we extended our investigation to other F508del-CFTR correctors, such as miglustat (Norez, 2006), an approved drug for Gaucher disease, in comparison with the galactose analogue NB-DGJ. " 01/01/2017 - "Lucerastat, an inhibitor of glucosylceramide synthase, has the potential to restore the balance between synthesis and degradation of glycosphingolipids in glycolipid storage disorders such as Gaucher disease and Fabry disease. "

Related Drugs and Biologics

1.	migalastat
2.	Biomarkers (Surrogate Marker)
3.	Galactose (Galactopyranose)
4.	ceramide glucosyltransferase
5.	miglustat (Zavesca)
6.	Pyrimethamine (Daraprim)
7.	Ambroxol (Mucosolvan)
8.	N-octyl-beta-valienamine
9.	Galactosidases
10.	Enzymes

Related Therapies and Procedures

1.	Enzyme Replacement Therapy
2.	Therapeutics
3.	Oral Administration