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 *

178 related articles for article (PubMed ID: 37036722)

  • 1. 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]  

  • 2. 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]  

  • 3. Enzyme replacement therapy for late-onset Pompe disease.
    Dalmia S; Sharma R; Ramaswami U; Hughes D; Jahnke N; Cole D; Smith S; Remmington T
    Cochrane Database Syst Rev; 2023 Dec; 12(12):CD012993. PubMed ID: 38084761
    [TBL] [Abstract][Full Text] [Related]  

  • 4. 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]  

  • 5. 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]  

  • 6. Effect of avalglucosidase alfa on disease-specific and general patient-reported outcomes in treatment-naïve adults with late-onset Pompe disease compared with alglucosidase alfa: Meaningful change analyses from the Phase 3 COMET trial.
    Toscano A; Pollissard L; Msihid J; van der Beek N; Kishnani PS; Dimachkie MM; Berger KI; DasMahapatra P; Thibault N; Hamed A; Zhou T; Haack KA; Schoser B
    Mol Genet Metab; 2024 Feb; 141(2):108121. PubMed ID: 38184428
    [TBL] [Abstract][Full Text] [Related]  

  • 7. 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]  

  • 8. 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]  

  • 9. Safety and efficacy of avalglucosidase alfa in individuals with infantile-onset Pompe disease enrolled in the phase 2, open-label Mini-COMET study: The 6-month primary analysis report.
    Kishnani PS; Kronn D; Brassier A; Broomfield A; Davison J; Hahn SH; Kumada S; Labarthe F; Ohki H; Pichard S; Prakalapakorn SG; Haack KA; Kittner B; Meng X; Sparks S; Wilson C; Zaher A; Chien YH;
    Genet Med; 2023 Feb; 25(2):100328. PubMed ID: 36542086
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial.
    Schoser B; Roberts M; Byrne BJ; Sitaraman S; Jiang H; Laforêt P; Toscano A; Castelli J; Díaz-Manera J; Goldman M; van der Ploeg AT; Bratkovic D; Kuchipudi S; Mozaffar T; Kishnani PS;
    Lancet Neurol; 2021 Dec; 20(12):1027-1037. PubMed ID: 34800400
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Applying the win ratio method in clinical trials of orphan drugs: an analysis of data from the COMET trial of avalglucosidase alfa in patients with late-onset Pompe disease.
    Boentert M; Berger KI; Díaz-Manera J; Dimachkie MM; Hamed A; Riou França L; Thibault N; Shukla P; Ishak J; Caro JJ
    Orphanet J Rare Dis; 2024 Jan; 19(1):14. PubMed ID: 38216959
    [TBL] [Abstract][Full Text] [Related]  

  • 12. 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]  

  • 13. Efficacy of avalglucosidase alfa on forced vital capacity percent predicted in treatment-naïve patients with late-onset Pompe disease: A pooled analysis of clinical trials.
    Mozaffar T; Riou França L; Msihid J; Shukla P; Proskorovsky I; Zhou T; Periquet M; An Haack K; Pollissard L; Straub V
    Mol Genet Metab Rep; 2024 Sep; 40():101109. PubMed ID: 39035044
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Post-hoc Nonparametric Analysis of Forced Vital Capacity in the COMET Trial Demonstrates Superiority of Avalglucosidase Alfa vs Alglucosidase Alfa.
    Boentert M; Campana ES; Attarian S; Diaz-Manera J; Dimachkie MM; Periquet M; Thibault N; Miossec P; Zhou T; Berger KI
    J Neuromuscul Dis; 2024; 11(2):369-374. PubMed ID: 38160363
    [TBL] [Abstract][Full Text] [Related]  

  • 15. 104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).
    Schoser B; Kishnani PS; Bratkovic D; Byrne BJ; Claeys KG; Díaz-Manera J; Laforêt P; Roberts M; Toscano A; van der Ploeg AT; Castelli J; Goldman M; Holdbrook F; Sitaraman Das S; Wasfi Y; Mozaffar T;
    J Neurol; 2024 May; 271(5):2810-2823. PubMed ID: 38418563
    [TBL] [Abstract][Full Text] [Related]  

  • 16. 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]  

  • 17. 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 Aug; 271(8):5433-5446. PubMed ID: 38896264
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study.
    Hahn SH; Kronn D; Leslie ND; Pena LDM; Tanpaiboon P; Gambello MJ; Gibson JB; Hillman R; Stockton DW; Day JW; Wang RY; An Haack K; Shafi R; Sparks S; Zhao Y; Wilson C; Kishnani PS;
    Genet Med; 2018 Oct; 20(10):1284-1294. PubMed ID: 29565424
    [TBL] [Abstract][Full Text] [Related]  

  • 19. A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe disease.
    Byrne BJ; Geberhiwot T; Barshop BA; Barohn R; Hughes D; Bratkovic D; Desnuelle C; Laforet P; Mengel E; Roberts M; Haroldsen P; Reilley K; Jayaram K; Yang K; Walsh L;
    Orphanet J Rare Dis; 2017 Aug; 12(1):144. PubMed ID: 28838325
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Enzyme replacement therapy for infantile-onset Pompe disease.
    Chen M; Zhang L; Quan S
    Cochrane Database Syst Rev; 2017 Nov; 11(11):CD011539. PubMed ID: 29155436
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 9.