BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

214 related articles for article (PubMed ID: 27489778)

  • 1. The influence of a polymorphism in the gene encoding angiotensin converting enzyme (ACE) on treatment outcomes in late-onset Pompe patients receiving alglucosidase alfa.
    Baek RC; Palmer R; Pomponio RJ; Lu Y; Ma X; McVie-Wylie AJ
    Mol Genet Metab Rep; 2016 Sep; 8():48-50. PubMed ID: 27489778
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Survival and long-term outcomes in late-onset Pompe disease following alglucosidase alfa treatment: a systematic review and meta-analysis.
    Schoser B; Stewart A; Kanters S; Hamed A; Jansen J; Chan K; Karamouzian M; Toscano A
    J Neurol; 2017 Apr; 264(4):621-630. PubMed ID: 27372449
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial.
    Diaz-Manera J; Kishnani PS; Kushlaf H; Ladha S; Mozaffar T; Straub V; Toscano A; van der Ploeg AT; Berger KI; Clemens PR; Chien YH; Day JW; Illarioshkin S; Roberts M; Attarian S; Borges JL; Bouhour F; Choi YC; Erdem-Ozdamar S; Goker-Alpan O; Kostera-Pruszczyk A; Haack KA; Hug C; Huynh-Ba O; Johnson J; Thibault N; Zhou T; Dimachkie MM; Schoser B;
    Lancet Neurol; 2021 Dec; 20(12):1012-1026. PubMed ID: 34800399
    [TBL] [Abstract][Full Text] [Related]  

  • 4. The ACE I/D polymorphism does not explain heterogeneity of natural course and response to enzyme replacement therapy in Pompe disease.
    Kuperus E; van der Meijden JC; In 't Groen SLM; Kroos MA; Hoogeveen-Westerveld M; Rizopoulos D; Martinez MYN; Kruijshaar ME; van Doorn PA; van der Beek NAME; van der Ploeg AT; Pijnappel WWMP
    PLoS One; 2018; 13(12):e0208854. PubMed ID: 30532252
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Real-world outcomes from a series of patients with late onset Pompe disease who switched from alglucosidase alfa to avalglucosidase alfa.
    Carter C; Boggs T; Case LE; Kishnani P
    Front Genet; 2024; 15():1309146. PubMed ID: 38313679
    [No Abstract]   [Full Text] [Related]  

  • 6. Real-life effectiveness 1 year after switching to avalglucosidase alfa in late-onset Pompe disease patients worsening on alglucosidase alfa therapy: A French cohort study.
    Tard C; Bouhour F; Michaud M; Beltran S; Fournier M; Demurger F; Lagrange E; Nollet S; Sacconi S; Noury JB; Magot A; Cintas P; Renard D; Deibener-Kaminsky J; Lefeuvre C; Davion JB; Salort-Campana E; Arrassi A; Taouagh N; Spinazzi M; ; Attarian S; Laforêt P
    Eur J Neurol; 2024 Jul; 31(7):e16292. PubMed ID: 38587143
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Respiratory function during enzyme replacement therapy in late-onset Pompe disease: longitudinal course, prognostic factors, and the impact of time from diagnosis to treatment start.
    Stockton DW; Kishnani P; van der Ploeg A; Llerena J; Boentert M; Roberts M; Byrne BJ; Araujo R; Maruti SS; Thibault N; Verhulst K; Berger KI
    J Neurol; 2020 Oct; 267(10):3038-3053. PubMed ID: 32524257
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study.
    van der Ploeg A; Carlier PG; Carlier RY; Kissel JT; Schoser B; Wenninger S; Pestronk A; Barohn RJ; Dimachkie MM; Goker-Alpan O; Mozaffar T; Pena LD; Simmons Z; Straub V; Guglieri M; Young P; Boentert M; Baudin PY; Wens S; Shafi R; Bjartmar C; Thurberg BL
    Mol Genet Metab; 2016 Sep; 119(1-2):115-23. PubMed ID: 27473031
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Clinical characteristics and genotypes in the ADVANCE baseline data set, a comprehensive cohort of US children and adolescents with Pompe disease.
    Kishnani PS; Gibson JB; Gambello MJ; Hillman R; Stockton DW; Kronn D; Leslie ND; Pena LDM; Tanpaiboon P; Day JW; Wang RY; Goldstein JL; An Haack K; Sparks SE; Zhao Y; Hahn SH;
    Genet Med; 2019 Nov; 21(11):2543-2551. PubMed ID: 31086307
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease.
    Dimachkie MM; Barohn RJ; Byrne B; Goker-Alpan O; Kishnani PS; Ladha S; Laforêt P; Mengel KE; Peña LDM; Sacconi S; Straub V; Trivedi J; Van Damme P; van der Ploeg AT; Vissing J; Young P; Haack KA; Foster M; Gilbert JM; Miossec P; Vitse O; Zhou T; Schoser B;
    Neurology; 2022 Aug; 99(5):e536-e548. PubMed ID: 35618441
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study.
    Pena LDM; Barohn RJ; Byrne BJ; Desnuelle C; Goker-Alpan O; Ladha S; Laforêt P; Mengel KE; Pestronk A; Pouget J; Schoser B; Straub V; Trivedi J; Van Damme P; Vissing J; Young P; Kacena K; Shafi R; Thurberg BL; Culm-Merdek K; van der Ploeg AT;
    Neuromuscul Disord; 2019 Mar; 29(3):167-186. PubMed ID: 30770310
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Pompe disease in Austria: clinical, genetic and epidemiological aspects.
    Löscher WN; Huemer M; Stulnig TM; Simschitz P; Iglseder S; Eggers C; Moser H; Möslinger D; Freilinger M; Lagler F; Grinzinger S; Reichhardt M; Bittner RE; Schmidt WM; Lex U; Brunner-Krainz M; Quasthoff S; Wanschitz JV
    J Neurol; 2018 Jan; 265(1):159-164. PubMed ID: 29181627
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Changes in forced vital capacity over ≤ 13 years among patients with late-onset Pompe disease treated with alglucosidase alfa: new modeling of real-world data from the Pompe Registry.
    Berger KI; Chien YH; Dubrovsky A; Kishnani PS; Llerena JC; Neilan E; Roberts M; Sheng B; Batista JL; Periquet M; Wilson KM; van der Ploeg AT
    J Neurol; 2024 Jun; ():. PubMed ID: 38896264
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa.
    van der Ploeg AT; Barohn R; Carlson L; Charrow J; Clemens PR; Hopkin RJ; Kishnani PS; Laforêt P; Morgan C; Nations S; Pestronk A; Plotkin H; Rosenbloom BE; Sims KB; Tsao E
    Mol Genet Metab; 2012 Nov; 107(3):456-61. PubMed ID: 23031366
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Newborn screening for Pompe disease in Italy: Long-term results and future challenges.
    Gragnaniello V; Pijnappel PWWM; Burlina AP; In 't Groen SLM; Gueraldi D; Cazzorla C; Maines E; Polo G; Salviati L; Di Salvo G; Burlina AB
    Mol Genet Metab Rep; 2022 Dec; 33():100929. PubMed ID: 36310651
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Albuterol as an adjunctive treatment to enzyme replacement therapy in infantile-onset Pompe disease.
    Chien YH; Hwu WL; Lee NC; Tsai FJ; Koeberl DD; Tsai WH; Chiu PC; Chang CL
    Mol Genet Metab Rep; 2017 Jun; 11():31-35. PubMed ID: 28480166
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Effects of short-to-long term enzyme replacement therapy (ERT) on skeletal muscle tissue in late onset Pompe disease (LOPD).
    Ripolone M; Violano R; Ronchi D; Mondello S; Nascimbeni A; Colombo I; Fagiolari G; Bordoni A; Fortunato F; Lucchini V; Saredi S; Filosto M; Musumeci O; Tonin P; Mongini T; Previtali S; Morandi L; Angelini C; Mora M; Sandri M; Sciacco M; Toscano A; Comi GP; Moggio M
    Neuropathol Appl Neurobiol; 2018 Aug; 44(5):449-462. PubMed ID: 28574618
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Earlier and higher dosing of alglucosidase alfa improve outcomes in patients with infantile-onset Pompe disease: Evidence from real-world experiences.
    Chien YH; Tsai WH; Chang CL; Chiu PC; Chou YY; Tsai FJ; Wong SL; Lee NC; Hwu WL
    Mol Genet Metab Rep; 2020 Jun; 23():100591. PubMed ID: 32373469
    [TBL] [Abstract][Full Text] [Related]  

  • 19. A molecular analysis of the GAA gene and clinical spectrum in 38 patients with Pompe disease in Japan.
    Fukuhara Y; Fuji N; Yamazaki N; Hirakiyama A; Kamioka T; Seo JH; Mashima R; Kosuga M; Okuyama T
    Mol Genet Metab Rep; 2018 Mar; 14():3-9. PubMed ID: 29124014
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial.
    Kishnani PS; Diaz-Manera J; Toscano A; Clemens PR; Ladha S; Berger KI; Kushlaf H; Straub V; Carvalho G; Mozaffar T; Roberts M; Attarian S; Chien YH; Choi YC; Day JW; Erdem-Ozdamar S; Illarioshkin S; Goker-Alpan O; Kostera-Pruszczyk A; van der Ploeg AT; An Haack K; Huynh-Ba O; Tammireddy S; Thibault N; Zhou T; Dimachkie MM; Schoser B;
    JAMA Neurol; 2023 Jun; 80(6):558-567. PubMed ID: 37036722
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 11.