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Journal Abstract Search

219 related items for PubMed ID: 37326923

  • 1. 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
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  • 2. 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 Sep; 104(1-2):118-22. PubMed ID: 21802969
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  • 3. 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 08; 18(1):108. PubMed ID: 37158969
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  • 4. 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, NEO1 Investigator Group.
    Neuromuscul Disord; 2019 Mar 08; 29(3):167-186. PubMed ID: 30770310
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  • 6. 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 08; 25(4):321-32. PubMed ID: 25617983
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  • 9. 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 08; 123(2):92-96. PubMed ID: 29289479
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  • 11. 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 08; 139(3):107611. PubMed ID: 37285781
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  • 12. 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 02; 17(1):31. PubMed ID: 35109913
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  • 13. Desensitization of an adult patient with Pompe disease and a history of anaphylaxis to alglucosidase alfa.
    Lipinski SE, Lipinski MJ, Burnette A, Platts-Mills TA, Wilson WG.
    Mol Genet Metab; 2009 Nov 02; 98(3):319-21. PubMed ID: 19640753
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  • 14. 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 27; 18(1):338. PubMed ID: 37891668
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  • 15. Effect of enzyme replacement therapy with alglucosidase alfa (Myozyme®) in 12 patients with advanced late-onset Pompe disease.
    Papadopoulos C, Orlikowski D, Prigent H, Lacour A, Tard C, Furby A, Praline J, Solé G, Hogrel JY, De Antonio M, Semplicini C, Deibener-Kaminsky J, Kaminsky P, Eymard B, Taouagh N, Perniconi B, Hamroun D, Laforêt P, French Pompe Study Group.
    Mol Genet Metab; 2017 Sep 27; 122(1-2):80-85. PubMed ID: 28648663
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  • 16. 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 27; 264(4):621-630. PubMed ID: 27372449
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  • 17. Alglucosidase alfa: new drug. Pompe disease: a short-term benefit.
    Prescrire Int; 2007 Dec 27; 16(92):240-1. PubMed ID: 18092404
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  • 19. 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 Dec 27; 15():1309146. PubMed ID: 38313679
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  • 20. 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 27; 107(3):456-61. PubMed ID: 23031366
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