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2. Improvement of bone mineral density after enzyme replacement therapy in Chinese late-onset Pompe disease patients. Sheng B, Chu YP, Wong WT, Yau EKC, Chen SPL, Luk WH. BMC Res Notes; 2017 Jul 28; 10(1):351. PubMed ID: 28754168 [Abstract] [Full Text] [Related]
3. Effects of short-to-long term enzyme replacement therapy (ERT) on skeletal muscle tissue in late onset Pompe disease (LOPD). Ripolone M, Violano R, Ronchi D, Mondello S, Nascimbeni A, Colombo I, Fagiolari G, Bordoni A, Fortunato F, Lucchini V, Saredi S, Filosto M, Musumeci O, Tonin P, Mongini T, Previtali S, Morandi L, Angelini C, Mora M, Sandri M, Sciacco M, Toscano A, Comi GP, Moggio M. Neuropathol Appl Neurobiol; 2018 Aug 28; 44(5):449-462. PubMed ID: 28574618 [Abstract] [Full Text] [Related]
4. 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 28; 120(3):163-172. PubMed ID: 28185884 [Abstract] [Full Text] [Related]
9. 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 28; 25(4):321-32. PubMed ID: 25617983 [Abstract] [Full Text] [Related]
10. Rare Variants in Autophagy and Non-Autophagy Genes in Late-Onset Pompe Disease: Suggestions of Their Disease-Modifying Role in Two Italian Families. Napolitano F, Bruno G, Terracciano C, Franzese G, Palomba NP, Scotto di Carlo F, Signoriello E, De Blasiis P, Navarro S, Gialluisi A, Melone MAB, Sampaolo S, Esposito T. Int J Mol Sci; 2021 Mar 31; 22(7):. PubMed ID: 33807278 [Abstract] [Full Text] [Related]
14. Quantitative computed tomography for enzyme replacement therapy in Pompe disease. Yonee C, Toyoshima M, Young SP, Maruyama S, Higuchi I, Narita A, Maegaki Y, Nanba E, Ohno K, Kawano Y. Brain Dev; 2012 Nov 31; 34(10):834-9. PubMed ID: 22521436 [Abstract] [Full Text] [Related]
16. Study of the effect of anti-rhGAA antibodies at low and intermediate titers in late onset Pompe patients treated with ERT. Fernández-Simón E, Carrasco-Rozas A, Gallardo E, González-Quereda L, Alonso-Pérez J, Belmonte I, Pedrosa-Hernández I, Montiel E, Segovia S, Suárez-Calvet X, Llauger J, Mayos M, Illa I, Barba-Romero MA, Barcena J, Paradas C, Carzorla MR, Creus C, Coll-Cantí J, Díaz M, Domínguez C, Fernández-Torrón R, García-Antelo MJ, Grau JM, López de Munáin A, Martínez-García FA, Morgado Y, Moreno A, Morís G, Muñoz-Blanco MA, Nascimento A, Parajuá-Pozo JL, Querol L, Rojas R, Robledo-Strauss A, Rojas-Marcos Í, Salazar JA, Usón M, Díaz-Manera J. Mol Genet Metab; 2019 Nov 31; 128(1-2):129-136. PubMed ID: 31378569 [Abstract] [Full Text] [Related]
17. 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 31; 122(1-2):80-85. PubMed ID: 28648663 [Abstract] [Full Text] [Related]
18. 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, European Pompe Consortium project group on classic infantile Pompe disease. Lancet Child Adolesc Health; 2022 Jan 31; 6(1):28-37. PubMed ID: 34822769 [Abstract] [Full Text] [Related]
19. Pompe disease in Austria: clinical, genetic and epidemiological aspects. Löscher WN, Huemer M, Stulnig TM, Simschitz P, Iglseder S, Eggers C, Moser H, Möslinger D, Freilinger M, Lagler F, Grinzinger S, Reichhardt M, Bittner RE, Schmidt WM, Lex U, Brunner-Krainz M, Quasthoff S, Wanschitz JV. J Neurol; 2018 Jan 31; 265(1):159-164. PubMed ID: 29181627 [Abstract] [Full Text] [Related]
20. 36 months observational clinical study of 38 adult Pompe disease patients under alglucosidase alfa enzyme replacement therapy. Regnery C, Kornblum C, Hanisch F, Vielhaber S, Strigl-Pill N, Grunert B, Müller-Felber W, Glocker FX, Spranger M, Deschauer M, Mengel E, Schoser B. J Inherit Metab Dis; 2012 Sep 31; 35(5):837-45. PubMed ID: 22290025 [Abstract] [Full Text] [Related] Page: [Next] [New Search]