A pharmacological chaperone improves memory by reducing Aβ and tau neuropathology in a mouse model with plaques and tangles. [PDF]
Li JG +4 more
europepmc +1 more source
Assessment of Gene Variant Amenability for Pharmacological Chaperone Therapy with 1-Deoxygalactonojirimycin in Fabry Disease. [PDF]
Lukas J +10 more
europepmc +1 more source
Synthesis of a C-2 Functionalized l-Iduronic Acid Derivative as a Candidate Pharmacological Chaperone for MPS II (Hunter Syndrome). [PDF]
Nagendra V +12 more
europepmc +1 more source
Two novel <i>GAA</i> missense variants and a known VUS in pompe disease: Functional characterization and pharmacological chaperone therapy. [PDF]
Xia Y +5 more
europepmc +1 more source
The sodium/glucose cotransporter 2 inhibitor empagliflozin is a pharmacological chaperone of cardiac Na<sub>v</sub>1.5 channels. [PDF]
Sauer J +12 more
europepmc +1 more source
Editorial Note: The Pharmacological Chaperone AT2220 Increases Recombinant Human Acid α-Glucosidase Uptake and Glycogen Reduction in a Mouse Model of Pompe Disease. [PDF]
PLOS One Editors.
europepmc +1 more source
Characterizing the inhibition of α-synuclein oligomerization by a pharmacological chaperone that prevents prion formation by the protein PrP. [PDF]
Dong C +4 more
europepmc +1 more source
In the last 20 or so years, significant advancements in our understanding of membrane protein structures have been driven by progress in cryo-electron microscopy, X-ray crystallography and in silico modeling.
Williams, Erin Quinn
core
In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease. [PDF]
Lukas J +5 more
europepmc +1 more source
Identification of Small Molecule Compounds for Pharmacological Chaperone Therapy of Aspartylglucosaminuria. [PDF]
Banning A +4 more
europepmc +1 more source

