BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

143 related articles for article (PubMed ID: 38802634)

  • 1. 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]  

  • 2. 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]  

  • 3. 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]  

  • 4. 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]  

  • 5. Early initiation of ambroxol treatment diminishes neurological manifestations of type 3 Gaucher disease: A long-term outcome of two siblings.
    Ramadža DP; Zekušić M; Žigman T; Škaričić A; Bogdanić A; Mustać G; Bošnjak-Nađ K; Ozretić D; Ohno K; Fumić K; Barić I
    Eur J Paediatr Neurol; 2021 May; 32():66-72. PubMed ID: 33836415
    [TBL] [Abstract][Full Text] [Related]  

  • 6. 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]  

  • 7. [A retrospective study on enzyme replacement therapy in patients with Gaucher disease].
    Duan YL; Zhang YH; Zang Y; Shi HP; Zhang WM; Hu YM
    Zhonghua Er Ke Za Zhi; 2006 Sep; 44(9):653-6. PubMed ID: 17217655
    [TBL] [Abstract][Full Text] [Related]  

  • 8. A multicenter, open-label extension study of velaglucerase alfa in Japanese patients with Gaucher disease: Results after a cumulative treatment period of 24months.
    Ida H; Tanaka A; Matsubayashi T; Murayama K; Hongo T; Lee HM; Mellgard B
    Blood Cells Mol Dis; 2016 Jul; 59():140-7. PubMed ID: 27241455
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Recombinant macrophage targeted enzyme replacement therapy for Gaucher disease in India.
    Nagral A; Mewawalla P; Jagadeesh S; Kabra M; Phadke SR; Verma IC; Puri RD; Gupta N; Kishnani PS; Mistry PK
    Indian Pediatr; 2011 Oct; 48(10):779-84. PubMed ID: 22080680
    [TBL] [Abstract][Full Text] [Related]  

  • 10. The appearance of newly identified intraocular lesions in Gaucher disease type 3 despite long-term glucocerebrosidase replacement therapy.
    Sawicka-Gutaj N; Machaczka M; Kulińska-Niedziela I; Bernardczyk-Meller J; Gutaj P; Sowiński J; Ruchała M
    Ups J Med Sci; 2016 Aug; 121(3):192-5. PubMed ID: 27064303
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Gaucher disease type 1 patients from the ICGG Gaucher Registry sustain initial clinical improvements during twenty years of imiglucerase treatment.
    Weinreb NJ; Camelo JS; Charrow J; McClain MR; Mistry P; Belmatoug N;
    Mol Genet Metab; 2021 Feb; 132(2):100-111. PubMed ID: 33485799
    [TBL] [Abstract][Full Text] [Related]  

  • 12. A phase 2 multi-center, open-label, switch-over trial to evaluate the safety and efficacy of Abcertin® in patients with type 1 Gaucher disease.
    Choi JH; Lee BH; Ko JM; Sohn YB; Lee JS; Kim GH; Heo SH; Park JY; Kim YM; Kim JH; Yoo HW
    J Korean Med Sci; 2015 Apr; 30(4):378-84. PubMed ID: 25829804
    [TBL] [Abstract][Full Text] [Related]  

  • 13. A Phase 3, multicenter, open-label, switchover trial to assess the safety and efficacy of taliglucerase alfa, a plant cell-expressed recombinant human glucocerebrosidase, in adult and pediatric patients with Gaucher disease previously treated with imiglucerase.
    Pastores GM; Petakov M; Giraldo P; Rosenbaum H; Szer J; Deegan PB; Amato DJ; Mengel E; Tan ES; Chertkoff R; Brill-Almon E; Zimran A
    Blood Cells Mol Dis; 2014 Dec; 53(4):253-60. PubMed ID: 24950666
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Long term effects of enzyme replacement therapy in an Italian cohort of type 3 Gaucher patients.
    Sechi A; Deroma L; Dardis A; Ciana G; Bertin N; Concolino D; Linari S; Perria C; Bembi B
    Mol Genet Metab; 2014 Nov; 113(3):213-8. PubMed ID: 25127542
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Eliglustat compared with imiglucerase in patients with Gaucher's disease type 1 stabilised on enzyme replacement therapy: a phase 3, randomised, open-label, non-inferiority trial.
    Cox TM; Drelichman G; Cravo R; Balwani M; Burrow TA; Martins AM; Lukina E; Rosenbloom B; Ross L; Angell J; Puga AC
    Lancet; 2015 Jun; 385(9985):2355-62. PubMed ID: 25819691
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Neurologic improvement in a type 3 Gaucher disease patient treated with imiglucerase/miglustat combination.
    Capablo JL; Franco R; de Cabezón AS; Alfonso P; Pocovi M; Giraldo P
    Epilepsia; 2007 Jul; 48(7):1406-8. PubMed ID: 17433057
    [TBL] [Abstract][Full Text] [Related]  

  • 17. 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]  

  • 18. Impact of imiglucerase supply shortage on clinical and laboratory parameters in Norrbottnian patients with Gaucher disease type 3.
    Machaczka M; Kämpe Björkvall C; Wieremiejczyk J; Paucar Arce M; Myhr-Eriksson K; Klimkowska M; Hägglund H; Svenningsson P
    Arch Immunol Ther Exp (Warsz); 2015 Feb; 63(1):65-71. PubMed ID: 25205209
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Long-term velaglucerase alfa treatment in children with Gaucher disease type 1 naïve to enzyme replacement therapy or previously treated with imiglucerase.
    Smith L; Rhead W; Charrow J; Shankar SP; Bavdekar A; Longo N; Mardach R; Harmatz P; Hangartner T; Lee HM; Crombez E; Pastores GM
    Mol Genet Metab; 2016 Feb; 117(2):164-71. PubMed ID: 26043810
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Thirty-year clinical outcomes after haematopoietic stem cell transplantation in neuronopathic Gaucher disease.
    Donald A; Björkvall CK; Vellodi A; ; Cox TM; Hughes D; Jones SA; Wynn R; Machaczka M
    Orphanet J Rare Dis; 2022 Jun; 17(1):234. PubMed ID: 35717194
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 8.