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Bioorganic & Medicinal Chemistry Letters|March 18, 2004
Small molecule biaryl FSH receptor agonists. Part 2: Lead optimization via parallel synthesisTao Guo, Anton E P Adang, Guizhen Dong, et al.
Plos One|March 9, 2013
Migalastat HCl reduces globotriaosylsphingosine (lyso-Gb3) in Fabry transgenic mice and in the plasma of Fabry patientsBrandy Young-Gqamana, Nastry Brignol, Hui-Hwa Chang, et al.
The FEBS Journal|February 13, 2010
The pharmacological chaperone isofagomine increases the activity of the Gaucher disease L444P mutant form of beta-glucosidaseRichie Khanna, Elfrida R Benjamin, Lee Pellegrino, et al.
Neuropharmacology|April 9, 2005
Pharmacological and pharmacokinetic characterization of the cannabinoid receptor 2 agonist, GW405833, utilizing rodent models of acute and chronic pain, anxiety, ataxia and catalepsyKenneth J Valenzano, Laykea Tafesse, Gary Lee, et al.
Bioorganic & Medicinal Chemistry Letters|March 18, 2004
Small molecule biaryl FSH receptor agonists. Part 1: Lead discovery via encoded combinatorial synthesisTao Guo, Anton E P Adang, Roland E Dolle, et al.
JCI Insight|March 8, 2019
Improved efficacy of a next-generation ERT in murine Pompe diseaseSu Xu, Yi Lun, Michelle Frascella, et al.
Plos One|August 8, 2015
Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused AgalsidaseDavid G Warnock, Daniel G Bichet, Myrl Holida, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|September 23, 2016
The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastatElfrida R Benjamin, Maria Cecilia Della Valle, Xiaoyang Wu, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|April 28, 2015
Coformulation of a Novel Human α-Galactosidase A With the Pharmacological Chaperone AT1001 Leads to Improved Substrate Reduction in Fabry MiceSu Xu, Yi Lun, Nastry Brignol, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|March 26, 2017
Duvoglustat HCl Increases Systemic and Tissue Exposure of Active Acid α-Glucosidase in Pompe Patients Co-administered with Alglucosidase αPriya Kishnani, Mark Tarnopolsky, Mark Roberts, et al.
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