✦ LIBER ✦

A pharmacogenetic analysis of Fabry patient responses to pharmacological chaperone treatment with AT1001 (migalastat hydrochloride)

✍ Scribed by Elfrida Benjamin; Xiaoyang Wu; M. Cecilia Della Valle; Evan Katz; Raphael Schiffmann; Jeffrey P. Castelli; Douglas S. Greene; Mathews Adera; Pol Boudes; David J. Lockhart; Kenneth J. Valenzano

Book ID: 116989121
Publisher: Elsevier Science
Year: 2011
Tongue: English
Weight: 59 KB
Volume: 102
Category: Article
ISSN: 1096-7192
DOI: 10.1016/j.ymgme.2010.11.023

No coin nor oath required. For personal study only.

📜 SIMILAR VOLUMES

A double-blind, randomized, placebo-cont

A double-blind, randomized, placebo-controlled study to evaluate the efficacy, safety and pharmacodynamics of AT1001 in patients with Fabry disease and AT1001-responsive GLA mutations

✍ Mathews Adera; Chemise Overton; Pol Boudes 📂 Article 📅 2011 🏛 Elsevier Science 🌐 English ⚖ 73 KB

Pharmacological chaperones aren't just f

Pharmacological chaperones aren't just for mutant enzymes anymore: Co-administration of AT1001 Stabilizes rh∂-Gal A, leading to greater uptake and substrate reduction in fabry patient-derived cells and GLA knockout mice

✍ Richie Khanna; Elfrida R. Benjamin; John J. Flanagan; Lee Pellegrino; Adriane Sc 📂 Article 📅 2011 🏛 Elsevier Science 🌐 English ⚖ 61 KB

A randomized, open-label study to compar

A randomized, open-label study to compare the efficacy and safety of AT1001 and enzyme replacement therapy (ERT) in patients with Fabry disease and AT1001-responsive GLA mutations, who were previously treated with ERT

✍ Mathews Adera; Allison Gladden; Pol Boudes 📂 Article 📅 2011 🏛 Elsevier Science 🌐 English ⚖ 60 KB

93. A Phase2 clinical trial to assess th

93. A Phase2 clinical trial to assess the safety and tolerability of the pharmacological chaperone AT 2101 in treatment-naïve adult patients with type I Gaucher disease

✍ Atul Mehta; Derralynn Hughes; Eugene Schneider; Quinn Dinh 📂 Article 📅 2010 🏛 Elsevier Science 🌐 English ⚖ 43 KB