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Journal Abstract Search

732 related items for PubMed ID: 28154884

  • 21. Gene Therapy for Pompe Disease: The Time is now.
    Colella P, Mingozzi F.
    Hum Gene Ther; 2019 Oct; 30(10):1245-1262. PubMed ID: 31298581
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  • 22. Adjunctive β2-agonist treatment reduces glycogen independently of receptor-mediated acid α-glucosidase uptake in the limb muscles of mice with Pompe disease.
    Farah BL, Madden L, Li S, Nance S, Bird A, Bursac N, Yen PM, Young SP, Koeberl DD.
    FASEB J; 2014 May; 28(5):2272-80. PubMed ID: 24448824
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  • 23. Design of Potent Mannose 6-Phosphate Analogues for the Functionalization of Lysosomal Enzymes To Improve the Treatment of Pompe Disease.
    El Cheikh K, Basile I, Da Silva A, Bernon C, Cérutti P, Salgues F, Perez M, Maynadier M, Gary-Bobo M, Caillaud C, Cérutti M, Garcia M, Morère A.
    Angew Chem Int Ed Engl; 2016 Nov 14; 55(47):14774-14777. PubMed ID: 27774736
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  • 30. The Release of a Soluble Glycosylated Protein from Glycogen by Recombinant Lysosomal α-Glucosidase (rhGAA) In Vitro and Its Presence in Serum In Vivo.
    Murray AK.
    Biomolecules; 2020 Nov 29; 10(12):. PubMed ID: 33260301
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  • 31. Aerobic training as an adjunctive therapy to enzyme replacement in Pompe disease.
    Nilsson MI, Samjoo IA, Hettinga BP, Koeberl DD, Zhang H, Hawke TJ, Nissar AA, Ali T, Brandt L, Ansari MU, Hazari H, Patel N, Amon J, Tarnopolsky MA.
    Mol Genet Metab; 2012 Nov 29; 107(3):469-79. PubMed ID: 23041258
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  • 32. A new assay for fast, reliable CRIM status determination in infantile-onset Pompe disease.
    Wang Z, Okamoto P, Keutzer J.
    Mol Genet Metab; 2014 Feb 29; 111(2):92-100. PubMed ID: 24044919
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  • 33. 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
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  • 34. The pharmacological chaperone AT2220 increases the specific activity and lysosomal delivery of mutant acid alpha-glucosidase, and promotes glycogen reduction in a transgenic mouse model of Pompe disease.
    Khanna R, Powe AC, Lun Y, Soska R, Feng J, Dhulipala R, Frascella M, Garcia A, Pellegrino LJ, Xu S, Brignol N, Toth MJ, Do HV, Lockhart DJ, Wustman BA, Valenzano KJ.
    PLoS One; 2014 Aug 24; 9(7):e102092. PubMed ID: 25036864
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  • 35. Autophagy and mistargeting of therapeutic enzyme in skeletal muscle in Pompe disease.
    Fukuda T, Ahearn M, Roberts A, Mattaliano RJ, Zaal K, Ralston E, Plotz PH, Raben N.
    Mol Ther; 2006 Dec 24; 14(6):831-9. PubMed ID: 17008131
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  • 36. Mapping the T helper cell response to acid α-glucosidase in Pompe mice.
    Nayak S, Sivakumar R, Cao O, Daniell H, Byrne BJ, Herzog RW.
    Mol Genet Metab; 2012 Jun 24; 106(2):189-95. PubMed ID: 22494547
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  • 37. Endolysosomal N-glycan processing is critical to attain the most active form of the enzyme acid alpha-glucosidase.
    Selvan N, Mehta N, Venkateswaran S, Brignol N, Graziano M, Sheikh MO, McAnany Y, Hung F, Madrid M, Krampetz R, Siano N, Mehta A, Brudvig J, Gotschall R, Weimer JM, Do HV.
    J Biol Chem; 2021 Jun 24; 296():100769. PubMed ID: 33971197
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  • 38. Dysregulation of multiple facets of glycogen metabolism in a murine model of Pompe disease.
    Taylor KM, Meyers E, Phipps M, Kishnani PS, Cheng SH, Scheule RK, Moreland RJ.
    PLoS One; 2013 Jun 24; 8(2):e56181. PubMed ID: 23457523
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  • 39. 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 24; 36(5):1177-1189. PubMed ID: 30879255
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