Abstract
N-Functionalized amino(hydroxymethyl)cyclopentanetriols are potent inhibitors of β-d-galactosidases and, for the first time, could be shown to act as pharmacological chaperones for GM1-gangliosidosis-associated lysosomal acid β-galactosidase thus representing a new structural type of pharmacological chaperones for this lysosomal storage disease.
Original language | English |
---|---|
Pages (from-to) | 3431-3435 |
Journal | Bioorganic & Medicinal Chemistry Letters |
Volume | 27 |
Issue number | 15 |
Publication status | Published - 2017 |