These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.


BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

282 related articles for article (PubMed ID: 36256599)

  • 1. 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]  

  • 2. Qualitative Study of the Patient Experience with Venglustat for Gaucher Disease Type 3 in a Phase 2 Open-Label, Multicenter, Multinational Study (LEAP).
    Schiffmann R; Mengel E; Wallace M; Rochmann C; Turnbull J; Krupnick R; Gwaltney C; Pulikottil-Jacob R; Batsu I; Zheng R; Hamed A
    Adv Ther; 2024 Jul; 41(7):2907-2923. PubMed ID: 38802634
    [TBL] [Abstract][Full Text] [Related]  

  • 3. 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]  

  • 4. 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]  

  • 5. Enzyme replacement and substrate reduction therapy for Gaucher disease.
    Shemesh E; Deroma L; Bembi B; Deegan P; Hollak C; Weinreb NJ; Cox TM
    Cochrane Database Syst Rev; 2015 Mar; 2015(3):CD010324. PubMed ID: 25812601
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Reductions in glucosylsphingosine (lyso-Gb1) in treatment-naïve and previously treated patients receiving velaglucerase alfa for type 1 Gaucher disease: Data from phase 3 clinical trials.
    Elstein D; Mellgard B; Dinh Q; Lan L; Qiu Y; Cozma C; Eichler S; Böttcher T; Zimran A
    Mol Genet Metab; 2017 Sep; 122(1-2):113-120. PubMed ID: 28851512
    [TBL] [Abstract][Full Text] [Related]  

  • 7. An observational study to investigate the relationship between plasma glucosylsphingosine (lyso-Gb1) concentration and treatment outcomes of patients with Gaucher disease in Japan.
    Ida H; Watanabe Y; Sagara R; Inoue Y; Fernandez J
    Orphanet J Rare Dis; 2022 Nov; 17(1):401. PubMed ID: 36329499
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Long-term hematological, visceral, and growth outcomes in children with Gaucher disease type 3 treated with imiglucerase in the International Collaborative Gaucher Group Gaucher Registry.
    El-Beshlawy A; Tylki-Szymanska A; Vellodi A; Belmatoug N; Grabowski GA; Kolodny EH; Batista JL; Cox GF; Mistry PK
    Mol Genet Metab; 2017; 120(1-2):47-56. PubMed ID: 28040394
    [TBL] [Abstract][Full Text] [Related]  

  • 9. 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]  

  • 10. Neuronopathic Gaucher disease in the mouse: viable combined selective saposin C deficiency and mutant glucocerebrosidase (V394L) mice with glucosylsphingosine and glucosylceramide accumulation and progressive neurological deficits.
    Sun Y; Liou B; Ran H; Skelton MR; Williams MT; Vorhees CV; Kitatani K; Hannun YA; Witte DP; Xu YH; Grabowski GA
    Hum Mol Genet; 2010 Mar; 19(6):1088-97. PubMed ID: 20047948
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Elevated plasma glucosylsphingosine in Gaucher disease: relation to phenotype, storage cell markers, and therapeutic response.
    Dekker N; van Dussen L; Hollak CE; Overkleeft H; Scheij S; Ghauharali K; van Breemen MJ; Ferraz MJ; Groener JE; Maas M; Wijburg FA; Speijer D; Tylki-Szymanska A; Mistry PK; Boot RG; Aerts JM
    Blood; 2011 Oct; 118(16):e118-27. PubMed ID: 21868580
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Safety and efficacy of venglustat in GBA1-associated Parkinson's disease: an international, multicentre, double-blind, randomised, placebo-controlled, phase 2 trial.
    Giladi N; Alcalay RN; Cutter G; Gasser T; Gurevich T; Höglinger GU; Marek K; Pacchetti C; Schapira AHV; Scherzer CR; Simuni T; Minini P; Sardi SP; Peterschmitt MJ
    Lancet Neurol; 2023 Aug; 22(8):661-671. PubMed ID: 37479372
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Optimization of Eliglustat-Based Glucosylceramide Synthase Inhibitors as Substrate Reduction Therapy for Gaucher Disease Type 3.
    Wilson MW; Shu L; Hinkovska-Galcheva V; Jin Y; Rajeswaran W; Abe A; Zhao T; Luo R; Wang L; Wen B; Liou B; Fannin V; Sun D; Sun Y; Shayman JA; Larsen SD
    ACS Chem Neurosci; 2020 Oct; 11(20):3464-3473. PubMed ID: 33035424
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Velaglucerase alfa for the management of type 1 Gaucher disease.
    Morris JL
    Clin Ther; 2012 Feb; 34(2):259-71. PubMed ID: 22264444
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Velaglucerase alfa, a human recombinant glucocerebrosidase enzyme replacement therapy for type 1 Gaucher disease.
    Pastores GM
    Curr Opin Investig Drugs; 2010 Apr; 11(4):472-8. PubMed ID: 20336596
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Plasma glucosylsphingosine correlations with baseline disease burden and response to eliglustat in two clinical trials of previously untreated adults with Gaucher disease type 1.
    Peterschmitt MJ; Foster MC; Ji AJ; Zajdel MB; Cox GF
    Mol Genet Metab; 2023 Mar; 138(3):107527. PubMed ID: 36739645
    [TBL] [Abstract][Full Text] [Related]  

  • 17. 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]  

  • 18. Osteopenia in Gaucher disease develops early in life: response to imiglucerase enzyme therapy in children, adolescents and adults.
    Mistry PK; Weinreb NJ; Kaplan P; Cole JA; Gwosdow AR; Hangartner T
    Blood Cells Mol Dis; 2011 Jan; 46(1):66-72. PubMed ID: 21112800
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Substrate reduction therapy of glycosphingolipid storage disorders.
    Aerts JM; Hollak CE; Boot RG; Groener JE; Maas M
    J Inherit Metab Dis; 2006; 29(2-3):449-56. PubMed ID: 16763917
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapy.
    Marshall J; McEachern KA; Chuang WL; Hutto E; Siegel CS; Shayman JA; Grabowski GA; Scheule RK; Copeland DP; Cheng SH
    J Inherit Metab Dis; 2010 Jun; 33(3):281-9. PubMed ID: 20336375
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 15.