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Journal Abstract Search

233 related items for PubMed ID: 36609019

  • 21. 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
    [Abstract] [Full Text] [Related]

  • 22. 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, POM-001/002 Investigators.
    Orphanet J Rare Dis; 2017 Aug 24; 12(1):144. PubMed ID: 28838325
    [Abstract] [Full Text] [Related]

  • 23. Evaluating avalglucosidase alfa for the management of late-onset Pompe disease.
    Angelini C.
    Expert Rev Neurother; 2024 Mar 24; 24(3):259-266. PubMed ID: 38261315
    [Abstract] [Full Text] [Related]

  • 24. The new era of Pompe disease: advances in the detection, understanding of the phenotypic spectrum, pathophysiology, and management.
    Kishnani PS, Beckemeyer AA, Mendelsohn NJ.
    Am J Med Genet C Semin Med Genet; 2012 Feb 15; 160C(1):1-7. PubMed ID: 22253049
    [Abstract] [Full Text] [Related]

  • 25. Optimizing treatment outcomes: immune tolerance induction in Pompe disease patients undergoing enzyme replacement therapy.
    Chen HA, Hsu RH, Fang CY, Desai AK, Lee NC, Hwu WL, Tsai FJ, Kishnani PS, Chien YH.
    Front Immunol; 2024 Feb 15; 15():1336599. PubMed ID: 38715621
    [Abstract] [Full Text] [Related]

  • 26. 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 15; 267(10):3038-3053. PubMed ID: 32524257
    [Abstract] [Full Text] [Related]

  • 27. New therapeutic approaches for Pompe disease: enzyme replacement therapy and beyond.
    Kishnani PS, Beckemeyer AA.
    Pediatr Endocrinol Rev; 2014 Sep 15; 12 Suppl 1():114-24. PubMed ID: 25345093
    [Abstract] [Full Text] [Related]

  • 28. Effects of enzyme replacement therapy on bone density in late onset Pompe disease.
    Avanti M, Martin A, Columbres RC, Mozaffar T, Kimonis V.
    Mol Genet Metab; 2023 Nov 15; 140(3):107644. PubMed ID: 37515933
    [Abstract] [Full Text] [Related]

  • 29. Comparing the efficacy of cipaglucosidase alfa plus miglustat with other enzyme replacement therapies for late-onset Pompe disease: a network meta-analysis utilizing patient-level and aggregate data.
    Shohet S, Hummel N, Fu S, Keyzor I, MacCulloch A, Johnson N, Castelli J, Czarny-Ozga I, Mozaffar T, Thom H.
    J Comp Eff Res; 2024 Oct 15; 13(10):e240045. PubMed ID: 39287071
    [Abstract] [Full Text] [Related]

  • 30. Significance of early diagnosis and treatment of adult late-onset Pompe disease on the effectiveness of enzyme replacement therapy in improving muscle strength and respiratory function: a case report.
    Mir M, Rouhani K, Rouhani K, Hassani M, Damirchi M, Yazdansetad S, Aghaei M.
    J Med Case Rep; 2024 Oct 08; 18(1):486. PubMed ID: 39375771
    [Abstract] [Full Text] [Related]

  • 31. 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 08; 271(8):5433-5446. PubMed ID: 38896264
    [Abstract] [Full Text] [Related]

  • 32. Enzyme replacement therapy and immunotherapy lead to significant functional improvement in two children with Pompe disease: a case report.
    Castellar-Leones SM, Ortiz-Corredor F, Manrique-Hernández D, Sánchez-Peñarete D, Ruiz-Ospina E, Soto-Peña D, Correa-Arrieta C.
    J Med Case Rep; 2024 Jul 18; 18(1):328. PubMed ID: 39020349
    [Abstract] [Full Text] [Related]

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  • 34. 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 18; 28(11):965-968. PubMed ID: 30314719
    [Abstract] [Full Text] [Related]

  • 35. 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 18; 36(5):1177-1189. PubMed ID: 30879255
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  • 37. Enzyme replacement therapy with alglucosidase alfa in 44 patients with late-onset glycogen storage disease type 2: 12-month results of an observational clinical trial.
    Strothotte S, Strigl-Pill N, Grunert B, Kornblum C, Eger K, Wessig C, Deschauer M, Breunig F, Glocker FX, Vielhaber S, Brejova A, Hilz M, Reiners K, Müller-Felber W, Mengel E, Spranger M, Schoser B.
    J Neurol; 2010 Jan 18; 257(1):91-7. PubMed ID: 19649685
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  • 39. Therapeutic thoroughfares for adults living with Pompe disease.
    Schoser B, Laforet P.
    Curr Opin Neurol; 2022 Oct 01; 35(5):645-650. PubMed ID: 35942661
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