Safety, Pharmacokinetics, and Pharmacodynamics of Oral Venglustat in Patients with Parkinson's Disease and a GBA Mutation: Results from Part 1 of the Randomized, Double-Blinded, Placebo-Controlled MOVES-PD Trial

Safety, Pharmacokinetics, and Pharmacodynamics of Oral Venglustat in Patients with Parkinson's Disease and a GBA Mutation: Results from Part 1 of the Randomized, Double-Blinded, Placebo-Controlled MOVES-PD Trial

Background: Glucocerebrosidase gene (GBA) mutations influence risk and prognosis of Parkinson's disease (PD), possibly through accumulation of glycosphingolipids, including glucosylceramide (GL-1). Venglustat is a novel, brain penetrant glucosylceramide synthase inhibitor. Objective: Evaluate v...

Full description

Bibliographic Details
Main Authors: Alcalay, R.N (Author), Cutter, G. (Author), Fischer, T.Z (Author), Gaemers, S.J.M (Author), Gasser, T. (Author), Giladi, N. (Author), Hatano, T. (Author), Hattori, N. (Author), Höglinger, G.U (Author), Isaacson, S.H (Author), Marek, K. (Author), Minini, P. (Author), Peterschmitt, M.J (Author), Saiki, H. (Author), Sardi, S.P (Author), Saubadu, S. (Author), Schapira, A.H.V (Author), Scherzer, C.R (Author), Sharma, J. (Author), Simuni, T. (Author), Walbillic, S. (Author)
Format: Article
Language:English
Published: IOS Press BV 2022
Subjects:
adult
area under the curve
Article
clearance at steady-state
clinical article
cohort analysis
confusion
connective tissue disease
controlled study
dose response
double blind procedure
drug blood level
drug cerebrospinal fluid level
drug dose escalation
drug megadose
drug safety
drug tolerability
drug withdrawal
ear disease
female
gastrointestinal disease
GBA-PD
gene mutation
glucocerebrosidase gene
glucocerebrosidase gene (GBA)
glucosylceramidase
glucosylceramide (GL-1)
glucosylceramide synthase (GCS) inhibition
heart murmur
heterozygote
human
infection
infestation
injury
inner ear disease
intoxication
Japanese (people)
kidney disease
low drug dose
male
maximum plasma concentration
MDS-Unified Parkinson Disease Rating Scale
mediastinum disease
mental disease
MOVES-PD
multicenter study
musculoskeletal disease
neurologic disease
panic
Parkinson disease
Parkinson's disease
pharmacodynamics
phase 2 clinical trial
placebo
post hoc analysis
randomized controlled trial
respiratory tract disease
skin disease
thorax disease
time to maximum plasma concentration
trough concentration
urinary tract disease
vascular disease
venglustat
Venglustat (GZ/SAR402671)
Online Access:View Fulltext in Publisher

Internet

View Fulltext in Publisher

Similar Items