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Journal Abstract Search

282 related items for PubMed ID: 30314719

  • 1. Enzyme replacement therapy with alglucosidase alfa in a late-onset Pompe disease patient during pregnancy.
    Oliveira Santos M, Evangelista T, Conceição I.
    Neuromuscul Disord; 2018 Nov; 28(11):965-968. PubMed ID: 30314719
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  • 3. First experience with enzyme replacement therapy during pregnancy and lactation in Pompe disease.
    de Vries JM, Brugma JD, Ozkan L, Steegers EA, Reuser AJ, van Doorn PA, van der Ploeg AT.
    Mol Genet Metab; 2011 Dec; 104(4):552-5. PubMed ID: 21967859
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  • 6. 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
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  • 7. 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
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  • 8. The emerging phenotype of late-onset Pompe disease: A systematic literature review.
    Chan J, Desai AK, Kazi ZB, Corey K, Austin S, Hobson-Webb LD, Case LE, Jones HN, Kishnani PS.
    Mol Genet Metab; 2017 Mar; 120(3):163-172. PubMed ID: 28185884
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  • 9. Treatment with enzyme replacement therapy during pregnancy in a patient with Pompe disease.
    Holbeck-Brendel M, Poulsen BK.
    Neuromuscul Disord; 2017 Oct; 27(10):956-958. PubMed ID: 28735900
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  • 14. White matter lesions in treated late onset Pompe disease are not different to matched controls.
    Schneider I, Hensel O, Zierz S.
    Mol Genet Metab; 2019 Jun; 127(2):128-131. PubMed ID: 31153821
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  • 15. Long-term benefit of enzyme replacement therapy with alglucosidase alfa in adults with Pompe disease: Prospective analysis from the French Pompe Registry.
    Semplicini C, De Antonio M, Taouagh N, Béhin A, Bouhour F, Echaniz-Laguna A, Magot A, Nadaj-Pakleza A, Orlikowski D, Sacconi S, Salort-Campana E, Solé G, Tard C, Zagnoli F, Hogrel JY, Hamroun D, Laforêt P, French Pompe Study Group.
    J Inherit Metab Dis; 2020 Nov; 43(6):1219-1231. PubMed ID: 32515844
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  • 18. Assessing the Role of Anti rh-GAA in Modulating Response to ERT in a Late-Onset Pompe Disease Cohort from the Italian GSDII Study Group.
    Filosto M, Cotti Piccinelli S, Ravaglia S, Servidei S, Moggio M, Musumeci O, Donati MA, Pegoraro E, Di Muzio A, Maggi L, Tonin P, Marrosu G, Sancricca C, Lerario A, Sacchini M, Semplicini C, Bozzoni V, Telese R, Bonanno S, Piras R, Maioli MA, Ricci G, Vercelli L, Galvagni A, Gallo Cassarino S, Caria F, Mongini T, Siciliano G, Padovani A, Toscano A.
    Adv Ther; 2019 May; 36(5):1177-1189. PubMed ID: 30879255
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  • 19. 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
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  • 20. New therapeutic approaches for Pompe disease: enzyme replacement therapy and beyond.
    Kishnani PS, Beckemeyer AA.
    Pediatr Endocrinol Rev; 2014 Sep; 12 Suppl 1():114-24. PubMed ID: 25345093
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