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 *

211 related articles for article (PubMed ID: 37158969)

  • 1. Home-based enzyme replacement therapy in children and adults with Pompe disease; a prospective study.
    Ditters IAM; van der Beek NAME; Brusse E; van der Ploeg AT; van den Hout JMP; Huidekoper HH
    Orphanet J Rare Dis; 2023 May; 18(1):108. PubMed ID: 37158969
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Home-Based Infusion of Alglucosidase Alfa Can Safely be Implemented in Adults with Late-Onset Pompe Disease: Lessons Learned from 18,380 Infusions.
    Ditters IAM; van Kooten HA; van der Beek NAME; Hardon JF; Ismailova G; Brusse E; Kruijshaar ME; van der Ploeg AT; van den Hout JMP; Huidekoper HH
    BioDrugs; 2023 Sep; 37(5):685-698. PubMed ID: 37326923
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Enzyme replacement therapy with alglucosidase alfa in Pompe disease: Clinical experience with rate escalation.
    Desai AK; Walters CK; Cope HL; Kazi ZB; DeArmey SM; Kishnani PS
    Mol Genet Metab; 2018 Feb; 123(2):92-96. PubMed ID: 29289479
    [TBL] [Abstract][Full Text] [Related]  

  • 4. An individually, modified approach to desensitize infants and young children with Pompe disease, and significant reactions to alglucosidase alfa infusions.
    El-Gharbawy AH; Mackey J; DeArmey S; Westby G; Grinnell SG; Malovrh P; Conway R; Kishnani PS
    Mol Genet Metab; 2011; 104(1-2):118-22. PubMed ID: 21802969
    [TBL] [Abstract][Full Text] [Related]  

  • 5. 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]  

  • 6. Effect of alglucosidase alfa dosage on survival and walking ability in patients with classic infantile Pompe disease: a multicentre observational cohort study from the European Pompe Consortium.
    Ditters IAM; Huidekoper HH; Kruijshaar ME; Rizopoulos D; Hahn A; Mongini TE; Labarthe F; Tardieu M; Chabrol B; Brassier A; Parini R; Parenti G; van der Beek NAME; van der Ploeg AT; van den Hout JMP;
    Lancet Child Adolesc Health; 2022 Jan; 6(1):28-37. PubMed ID: 34822769
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease.
    Case LE; Bjartmar C; Morgan C; Casey R; Charrow J; Clancy JP; Dasouki M; DeArmey S; Nedd K; Nevins M; Peters H; Phillips D; Spigelman Z; Tifft C; Kishnani PS
    Neuromuscul Disord; 2015 Apr; 25(4):321-32. PubMed ID: 25617983
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease.
    Kanters TA; van der Ploeg AT; Kruijshaar ME; Rizopoulos D; Redekop WK; Rutten-van Mӧlken MPMH; Hakkaart-van Roijen L
    Orphanet J Rare Dis; 2017 Dec; 12(1):179. PubMed ID: 29237491
    [TBL] [Abstract][Full Text] [Related]  

  • 9. High antibody titer in an adult with Pompe disease affects treatment with alglucosidase alfa.
    de Vries JM; van der Beek NA; Kroos MA; Ozkan L; van Doorn PA; Richards SM; Sung CC; Brugma JD; Zandbergen AA; van der Ploeg AT; Reuser AJ
    Mol Genet Metab; 2010 Dec; 101(4):338-45. PubMed ID: 20826098
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Antibodies against recombinant human alpha-glucosidase do not seem to affect clinical outcome in childhood onset Pompe disease.
    van Kooten HA; Ditters IAM; Hoogeveen-Westerveld M; Jacobs EH; van den Hout JMP; van Doorn PA; Pijnappel WWMP; van der Ploeg AT; van der Beek NAME
    Orphanet J Rare Dis; 2022 Feb; 17(1):31. PubMed ID: 35109913
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease.
    Kanters TA; Hoogenboom-Plug I; Rutten-Van Mölken MP; Redekop WK; van der Ploeg AT; Hakkaart L
    Orphanet J Rare Dis; 2014 May; 9():75. PubMed ID: 24884717
    [TBL] [Abstract][Full Text] [Related]  

  • 12. 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]  

  • 13. Safety outcomes and patients' preferences for home-based intravenous enzyme replacement therapy (ERT) in pompe disease and mucopolysaccharidosis type I (MPS I) disorder: COVID-19 and beyond.
    Toscano A; Musumeci O; Sacchini M; Ravaglia S; Siciliano G; Fiumara A; Verrecchia E; Maione M; Gentile J; Fischetto R; Crescimanno G; Taurisano R; Sechi A; Gasperini S; Cianci V; Maggi L; Parini R; Lupica A; Scarpa M
    Orphanet J Rare Dis; 2023 Oct; 18(1):338. PubMed ID: 37891668
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Ten years of the international Pompe survey: patient reported outcomes as a reliable tool for studying treated and untreated children and adults with non-classic Pompe disease.
    van der Meijden JC; Güngör D; Kruijshaar ME; Muir AD; Broekgaarden HA; van der Ploeg AT
    J Inherit Metab Dis; 2015 May; 38(3):495-503. PubMed ID: 25112389
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Effects of higher and more frequent dosing of alglucosidase alfa and immunomodulation on long-term clinical outcome of classic infantile Pompe patients.
    Poelman E; van den Dorpel JJA; Hoogeveen-Westerveld M; van den Hout JMP; van der Giessen LJ; van der Beek NAME; Pijnappel WWMP; van der Ploeg AT
    J Inherit Metab Dis; 2020 Nov; 43(6):1243-1253. PubMed ID: 32506446
    [TBL] [Abstract][Full Text] [Related]  

  • 16. European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10-year experience.
    van der Ploeg AT; Kruijshaar ME; Toscano A; Laforêt P; Angelini C; Lachmann RH; Pascual Pascual SI; Roberts M; Rösler K; Stulnig T; van Doorn PA; Van den Bergh PYK; Vissing J; Schoser B;
    Eur J Neurol; 2017 Jun; 24(6):768-e31. PubMed ID: 28477382
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center study.
    van Gelder CM; Poelman E; Plug I; Hoogeveen-Westerveld M; van der Beek NAME; Reuser AJJ; van der Ploeg AT
    J Inherit Metab Dis; 2016 May; 39(3):383-390. PubMed ID: 26768149
    [TBL] [Abstract][Full Text] [Related]  

  • 18. 36-Months follow-up assessment after cessation and resuming of enzyme replacement therapy in late onset Pompe disease: data from the Swiss Pompe Registry.
    Scheidegger O; Leupold D; Sauter R; Findling O; Rösler KM; Hundsberger T
    J Neurol; 2018 Dec; 265(12):2783-2788. PubMed ID: 30232608
    [TBL] [Abstract][Full Text] [Related]  

  • 19. STIG study: real-world data of long-term outcomes of adults with Pompe disease under enzyme replacement therapy with alglucosidase alfa.
    Gutschmidt K; Musumeci O; Díaz-Manera J; Chien YH; Knop KC; Wenninger S; Montagnese F; Pugliese A; Tavilla G; Alonso-Pérez J; Hwu PW; Toscano A; Schoser B
    J Neurol; 2021 Jul; 268(7):2482-2492. PubMed ID: 33543425
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Hypersensitivity infusion-associated reactions induced by enzyme replacement therapy in a cohort of patients with late-onset Pompe disease: An experience from the French Pompe Registry.
    Lessard LER; Tard C; Salort-Campana E; Sacconi S; Béhin A; Bassez G; Orlikowski D; Merle P; Nollet S; Gallay L; Bérard F; Robinson P; Bouhour F; Laforêt P
    Mol Genet Metab; 2023 Jul; 139(3):107611. PubMed ID: 37285781
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 11.