The drug candidate is administered orally as a capsule. The drug candidate targets glucosylceramide synthase (GCS). It is a new molecular entity (NME). It was under development for Parkinson's disease ...

Department of Chemistry, University of Florida, Gainesville, Florida 32611, United States ...

Revised: This Reviewed Preprint has been revised by the authors in response to the previous round of peer review; the eLife assessment and the public reviews have been updated where necessary by the ...

The low-density lipoprotein receptor (LDLR) is the primary mechanism for uptake of cholesterol-carrying particles into cells. The region of the LDLR implicated in receptor recycling and ...