BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

303 related articles for article (PubMed ID: 26948439)

  • 1. 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]  

  • 2. 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]  

  • 3. 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]  

  • 4. Systemic delivery of a glucosylceramide synthase inhibitor reduces CNS substrates and increases lifespan in a mouse model of type 2 Gaucher disease.
    Cabrera-Salazar MA; Deriso M; Bercury SD; Li L; Lydon JT; Weber W; Pande N; Cromwell MA; Copeland D; Leonard J; Cheng SH; Scheule RK
    PLoS One; 2012; 7(8):e43310. PubMed ID: 22912851
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Substrate reduction therapy using Genz-667161 reduces levels of pathogenic components in a mouse model of neuronopathic forms of Gaucher disease.
    Blumenreich S; Yaacobi C; Vardi A; Barav OB; Vitner EB; Park H; Wang B; Cheng SH; Sardi SP; Futerman AH
    J Neurochem; 2021 Mar; 156(5):692-701. PubMed ID: 32743826
    [TBL] [Abstract][Full Text] [Related]  

  • 6. A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease.
    McEachern KA; Fung J; Komarnitsky S; Siegel CS; Chuang WL; Hutto E; Shayman JA; Grabowski GA; Aerts JM; Cheng SH; Copeland DP; Marshall J
    Mol Genet Metab; 2007 Jul; 91(3):259-67. PubMed ID: 17509920
    [TBL] [Abstract][Full Text] [Related]  

  • 7. 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]  

  • 8. Glucosylceramide synthase inhibition alleviates aberrations in synucleinopathy models.
    Sardi SP; Viel C; Clarke J; Treleaven CM; Richards AM; Park H; Olszewski MA; Dodge JC; Marshall J; Makino E; Wang B; Sidman RL; Cheng SH; Shihabuddin LS
    Proc Natl Acad Sci U S A; 2017 Mar; 114(10):2699-2704. PubMed ID: 28223512
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Viral delivery of a microRNA to Gba to the mouse central nervous system models neuronopathic Gaucher disease.
    Jackson KL; Viel C; Clarke J; Bu J; Chan M; Wang B; Shihabuddin LS; Sardi SP
    Neurobiol Dis; 2019 Oct; 130():104513. PubMed ID: 31233883
    [TBL] [Abstract][Full Text] [Related]  

  • 10. A new brain-penetrant glucosylceramide synthase inhibitor as potential Therapeutics for Gaucher disease.
    Fujii T; Tanaka Y; Oki H; Sato S; Shibata S; Maru T; Tanaka Y; Tanaka M; Onishi T
    J Neurochem; 2021 Nov; 159(3):543-553. PubMed ID: 34398463
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Iminosugar-based inhibitors of glucosylceramide synthase prolong survival but paradoxically increase brain glucosylceramide levels in Niemann-Pick C mice.
    Nietupski JB; Pacheco JJ; Chuang WL; Maratea K; Li L; Foley J; Ashe KM; Cooper CG; Aerts JM; Copeland DP; Scheule RK; Cheng SH; Marshall J
    Mol Genet Metab; 2012 Apr; 105(4):621-8. PubMed ID: 22366055
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Substrate reduction therapy: clinical evaluation in type 1 Gaucher disease.
    Moyses C
    Philos Trans R Soc Lond B Biol Sci; 2003 May; 358(1433):955-60. PubMed ID: 12803929
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Eliglustat tartrate for the treatment of adults with type 1 Gaucher disease.
    Bennett LL; Turcotte K
    Drug Des Devel Ther; 2015; 9():4639-47. PubMed ID: 26345314
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Combination therapy in a patient with chronic neuronopathic Gaucher disease: a case report.
    Ceravolo F; Grisolia M; Sestito S; Falvo F; Moricca MT; Concolino D
    J Med Case Rep; 2017 Jan; 11(1):19. PubMed ID: 28103924
    [TBL] [Abstract][Full Text] [Related]  

  • 15. A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1.
    Lukina E; Watman N; Arreguin EA; Banikazemi M; Dragosky M; Iastrebner M; Rosenbaum H; Phillips M; Pastores GM; Rosenthal DI; Kaper M; Singh T; Puga AC; Bonate PL; Peterschmitt MJ
    Blood; 2010 Aug; 116(6):893-9. PubMed ID: 20439622
    [TBL] [Abstract][Full Text] [Related]  

  • 16. 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]  

  • 17. Inhibition of UDP-glucosylceramide synthase in mice prevents Gaucher disease-associated B-cell malignancy.
    Pavlova EV; Archer J; Wang S; Dekker N; Aerts JM; Karlsson S; Cox TM
    J Pathol; 2015 Jan; 235(1):113-24. PubMed ID: 25256118
    [TBL] [Abstract][Full Text] [Related]  

  • 18. 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]  

  • 19. 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]  

  • 20. 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]  

    [Next]    [New Search]
    of 16.