Detalles de la búsqueda
1.
Duvoglustat HCl Increases Systemic and Tissue Exposure of Active Acid α-Glucosidase in Pompe Patients Co-administered with Alglucosidase α.
Mol Ther;
25(5): 1199-1208, 2017 05 03.
Artículo
en Inglés
| MEDLINE | ID: mdl-28341561
2.
Glucosylceramide and Glucosylsphingosine Quantitation by Liquid Chromatography-Tandem Mass Spectrometry to Enable In Vivo Preclinical Studies of Neuronopathic Gaucher Disease.
Anal Chem;
89(16): 8288-8295, 2017 08 15.
Artículo
en Inglés
| MEDLINE | ID: mdl-28686011
3.
The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat.
Genet Med;
19(4): 430-438, 2017 04.
Artículo
en Inglés
| MEDLINE | ID: mdl-27657681
4.
Pharmacological Chaperone Therapy: Preclinical Development, Clinical Translation, and Prospects for the Treatment of Lysosomal Storage Disorders.
Mol Ther;
23(7): 1138-1148, 2015 Jul.
Artículo
en Inglés
| MEDLINE | ID: mdl-25881001
5.
Coformulation of a Novel Human α-Galactosidase A With the Pharmacological Chaperone AT1001 Leads to Improved Substrate Reduction in Fabry Mice.
Mol Ther;
23(7): 1169-1181, 2015 Jul.
Artículo
en Inglés
| MEDLINE | ID: mdl-25915924
6.
Correction of lysosomal dysfunction as a therapeutic strategy for neurodegenerative diseases.
Bioorg Med Chem Lett;
24(14): 3001-5, 2014 Jul 15.
Artículo
en Inglés
| MEDLINE | ID: mdl-24894562
7.
Lysosomal dysfunction in a mouse model of Sandhoff disease leads to accumulation of ganglioside-bound amyloid-ß peptide.
J Neurosci;
32(15): 5223-36, 2012 Apr 11.
Artículo
en Inglés
| MEDLINE | ID: mdl-22496568
8.
Co-administration with the pharmacological chaperone AT1001 increases recombinant human α-galactosidase A tissue uptake and improves substrate reduction in Fabry mice.
Mol Ther;
20(4): 717-26, 2012 Apr.
Artículo
en Inglés
| MEDLINE | ID: mdl-22215019
9.
A pharmacogenetic approach to identify mutant forms of α-galactosidase A that respond to a pharmacological chaperone for Fabry disease.
Hum Mutat;
32(8): 965-77, 2011 Aug.
Artículo
en Inglés
| MEDLINE | ID: mdl-21598360
10.
The pharmacological chaperone 1-deoxygalactonojirimycin reduces tissue globotriaosylceramide levels in a mouse model of Fabry disease.
Mol Ther;
18(1): 23-33, 2010 Jan.
Artículo
en Inglés
| MEDLINE | ID: mdl-19773742
11.
Pharmacological chaperones restore function to MC4R mutants responsible for severe early-onset obesity.
J Pharmacol Exp Ther;
335(3): 520-32, 2010 Dec.
Artículo
en Inglés
| MEDLINE | ID: mdl-20826565
12.
The pharmacological chaperone 1-deoxynojirimycin increases the activity and lysosomal trafficking of multiple mutant forms of acid alpha-glucosidase.
Hum Mutat;
30(12): 1683-92, 2009 Dec.
Artículo
en Inglés
| MEDLINE | ID: mdl-19862843
13.
Improved efficacy of a next-generation ERT in murine Pompe disease.
JCI Insight;
4(5)2019 03 07.
Artículo
en Inglés
| MEDLINE | ID: mdl-30843882
14.
State-dependent compound inhibition of Nav1.2 sodium channels using the FLIPR Vm dye: on-target and off-target effects of diverse pharmacological agents.
J Biomol Screen;
11(1): 29-39, 2006 Feb.
Artículo
en Inglés
| MEDLINE | ID: mdl-16234337
15.
Pharmacological characterization of recombinant N-type calcium channel (Cav2.2) mediated calcium mobilization using FLIPR.
Biochem Pharmacol;
72(6): 770-82, 2006 Sep 14.
Artículo
en Inglés
| MEDLINE | ID: mdl-16844100
16.
Pharmacological and pharmacokinetic characterization of the cannabinoid receptor 2 agonist, GW405833, utilizing rodent models of acute and chronic pain, anxiety, ataxia and catalepsy.
Neuropharmacology;
48(5): 658-72, 2005 Apr.
Artículo
en Inglés
| MEDLINE | ID: mdl-15814101
17.
Validation of a fluorescent imaging plate reader membrane potential assay for high-throughput screening of glycine transporter modulators.
J Biomol Screen;
10(4): 365-73, 2005 Jun.
Artículo
en Inglés
| MEDLINE | ID: mdl-15964938
18.
A role for cannabinoid receptors, but not endogenous opioids, in the antinociceptive activity of the CB2-selective agonist, GW405833.
Eur J Pharmacol;
528(1-3): 65-72, 2005 Dec 28.
Artículo
en Inglés
| MEDLINE | ID: mdl-16316650
19.
Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase.
PLoS One;
10(8): e0134341, 2015.
Artículo
en Inglés
| MEDLINE | ID: mdl-26252393
20.
Novel, potent ORL-1 receptor agonist peptides containing alpha-Helix-promoting conformational constraints.
J Med Chem;
45(24): 5280-6, 2002 Nov 21.
Artículo
en Inglés
| MEDLINE | ID: mdl-12431054