BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

209 related articles for article (PubMed ID: 32851809)

  • 1. Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Oral Venglustat in Healthy Volunteers.
    Peterschmitt MJ; Crawford NPS; Gaemers SJM; Ji AJ; Sharma J; Pham TT
    Clin Pharmacol Drug Dev; 2021 Jan; 10(1):86-98. PubMed ID: 32851809
    [TBL] [Abstract][Full Text] [Related]  

  • 2. 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.
    Peterschmitt MJ; Saiki H; Hatano T; Gasser T; Isaacson SH; Gaemers SJM; Minini P; Saubadu S; Sharma J; Walbillic S; Alcalay RN; Cutter G; Hattori N; Höglinger GU; Marek K; Schapira AHV; Scherzer CR; Simuni T; Giladi N; Sardi SP; Fischer TZ;
    J Parkinsons Dis; 2022; 12(2):557-570. PubMed ID: 34897099
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Pharmacokinetics, Safety, and Tolerability of Single-Dose Orally Administered Venglustat in Healthy Chinese Volunteers.
    Li Y; Li Y; Li L; Xue W; Xin K; Wang T; Shi A
    Clin Drug Investig; 2023 Jun; 43(6):413-420. PubMed ID: 37269489
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Phase 1 Healthy Volunteer Study of AL01211, an Oral, Non-brain Penetrant Glucosylceramide Synthase Inhibitor, to Treat Fabry Disease and Type 1 Gaucher Disease.
    Babcock M; Zheng J; Gail Shurr J; Li L; Wang B; Huertas P; Ryan PJ; Shen Y; Garovoy M
    Clin Pharmacol Drug Dev; 2024 Jun; 13(6):696-709. PubMed ID: 38363061
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Venglustat combined with imiglucerase for neurological disease in adults with Gaucher disease type 3: the LEAP trial.
    Schiffmann R; Cox TM; Dedieu JF; Gaemers SJM; Hennermann JB; Ida H; Mengel E; Minini P; Mistry P; Musholt PB; Scott D; Sharma J; Peterschmitt MJ
    Brain; 2023 Feb; 146(2):461-474. PubMed ID: 36256599
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Oral AL01211 in Healthy Chinese Volunteers.
    Dong L; Xiang J; Babcock M; Cheng Y; Wang Y; Shen Y; Li L; Tan L; Garovoy M; Hu W; Zheng J
    Clin Drug Investig; 2024 Jun; 44(6):387-398. PubMed ID: 38698285
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Venglustat, an orally administered glucosylceramide synthase inhibitor: Assessment over 3 years in adult males with classic Fabry disease in an open-label phase 2 study and its extension study.
    Deegan PB; Goker-Alpan O; Geberhiwot T; Hopkin RJ; Lukina E; Tylki-Szymanska A; Zaher A; Sensinger C; Gaemers SJM; Modur V; Thurberg BL; Sharma J; Najafian B; Mauer M; DasMahapatra P; Wilcox WR; Germain DP
    Mol Genet Metab; 2023 Feb; 138(2):106963. PubMed ID: 36481125
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Safety, tolerability, and pharmacokinetics of eliglustat tartrate (Genz-112638) after single doses, multiple doses, and food in healthy volunteers.
    Peterschmitt MJ; Burke A; Blankstein L; Smith SE; Puga AC; Kramer WG; Harris JA; Mathews D; Bonate PL
    J Clin Pharmacol; 2011 May; 51(5):695-705. PubMed ID: 20864621
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Preclinical pharmacology of glucosylceramide synthase inhibitor venglustat in a GBA-related synucleinopathy model.
    Viel C; Clarke J; Kayatekin C; Richards AM; Chiang MSR; Park H; Wang B; Shihabuddin LS; Sardi SP
    Sci Rep; 2021 Oct; 11(1):20945. PubMed ID: 34686711
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease.
    Ashe KM; Budman E; Bangari DS; Siegel CS; Nietupski JB; Wang B; Desnick RJ; Scheule RK; Leonard JP; Cheng SH; Marshall J
    Mol Med; 2015 Apr; 21(1):389-99. PubMed ID: 25938659
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Venglustat, a Novel Glucosylceramide Synthase Inhibitor, in Patients at Risk of Rapidly Progressing ADPKD: Primary Results of a Double-Blind, Placebo-Controlled, Phase 2/3 Randomized Clinical Trial.
    Gansevoort RT; Hariri A; Minini P; Ahn C; Chapman AB; Horie S; Knebelmann B; Mrug M; Ong ACM; Pei YPC; Torres VE; Modur V; Antonshchuk I; Perrone RD
    Am J Kidney Dis; 2023 May; 81(5):517-527.e1. PubMed ID: 36535535
    [TBL] [Abstract][Full Text] [Related]  

  • 12. First-in-human single-dose study of nizubaglustat, a dual inhibitor of ceramide glucosyltransferase and non-lysosomal glucosylceramidase: Safety, tolerability, pharmacokinetics, and pharmacodynamics of single ascending and multiple doses in healthy adults.
    Paquet Luzy C; Doppler E; Polasek TM; Giorgino R
    Mol Genet Metab; 2024 Jan; 141(1):108113. PubMed ID: 38113551
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Pharmacodynamics, pharmacokinetics, safety, and tolerability of encenicline, a selective α7 nicotinic receptor partial agonist, in single ascending-dose and bioavailability studies.
    Barbier AJ; Hilhorst M; Van Vliet A; Snyder P; Palfreyman MG; Gawryl M; Dgetluck N; Massaro M; Tiessen R; Timmerman W; Hilt DC
    Clin Ther; 2015 Feb; 37(2):311-24. PubMed ID: 25438724
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Substrate Reduction Therapy for Sandhoff Disease through Inhibition of Glucosylceramide Synthase Activity.
    Marshall J; Nietupski JB; Park H; Cao J; Bangari DS; Silvescu C; Wilper T; Randall K; Tietz D; Wang B; Ying X; Leonard JP; Cheng SH
    Mol Ther; 2019 Aug; 27(8):1495-1506. PubMed ID: 31208914
    [TBL] [Abstract][Full Text] [Related]  

  • 15. CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease.
    Marshall J; Sun Y; Bangari DS; Budman E; Park H; Nietupski JB; Allaire A; Cromwell MA; Wang B; Grabowski GA; Leonard JP; Cheng SH
    Mol Ther; 2016 Jun; 24(6):1019-1029. PubMed ID: 26948439
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Impact of the organic cation transporter 2 inhibitor cimetidine on the single-dose pharmacokinetics of the glucosylceramide synthase inhibitor lucerastat in healthy subjects.
    Boof ML; Halabi A; Ufer M; Dingemanse J
    Eur J Clin Pharmacol; 2020 Mar; 76(3):431-437. PubMed ID: 31836927
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Assessment of Target Engagement in a First-in-Human Trial with Sinbaglustat, an Iminosugar to Treat Lysosomal Storage Disorders.
    Gehin M; Melchior M; Welford RWD; Sidharta PN; Dingemanse J
    Clin Transl Sci; 2021 Mar; 14(2):558-567. PubMed ID: 33142037
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Safety, Tolerability and Pharmacokinetics of FAAH Inhibitor V158866: A Double-Blind, Randomised, Placebo-Controlled Phase I Study in Healthy Volunteers.
    Pawsey S; Wood M; Browne H; Donaldson K; Christie M; Warrington S
    Drugs R D; 2016 Jun; 16(2):181-91. PubMed ID: 26987975
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Lucerastat, an iminosugar with potential as substrate reduction therapy for glycolipid storage disorders: safety, tolerability, and pharmacokinetics in healthy subjects.
    Guérard N; Morand O; Dingemanse J
    Orphanet J Rare Dis; 2017 Jan; 12(1):9. PubMed ID: 28088251
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Pharmacokinetics, safety, and tolerability of GLPG0259, a mitogen-activated protein kinase-activated protein kinase 5 (MAPKAPK5) inhibitor, given as single and multiple doses to healthy male subjects.
    Namour F; Vanhoutte FP; Beetens J; Blockhuys S; De Weer M; Wigerinck P
    Drugs R D; 2012 Sep; 12(3):141-63. PubMed ID: 22950522
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 11.