170 related articles for article (PubMed ID: 37419682)
21. 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; 44(5):449-462. PubMed ID: 28574618
[TBL] [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;
Orphanet J Rare Dis; 2017 Aug; 12(1):144. PubMed ID: 28838325
[TBL] [Abstract][Full Text] [Related]
23. STIG study: real-world data of long-term outcomes of adults with Pompe disease under enzyme replacement therapy with alglucosidase alfa.
Gutschmidt K; Musumeci O; Díaz-Manera J; Chien YH; Knop KC; Wenninger S; Montagnese F; Pugliese A; Tavilla G; Alonso-Pérez J; Hwu PW; Toscano A; Schoser B
J Neurol; 2021 Jul; 268(7):2482-2492. PubMed ID: 33543425
[TBL] [Abstract][Full Text] [Related]
24. [Pompe disease treated with enzyme replacement therapy in pregnancy].
Grosz Z; Várdi KV; Molnár JM
Ideggyogy Sz; 2020 Sep; 73(9-10):339-344. PubMed ID: 33035415
[TBL] [Abstract][Full Text] [Related]
25. Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial.
Schoser B; Roberts M; Byrne BJ; Sitaraman S; Jiang H; Laforêt P; Toscano A; Castelli J; Díaz-Manera J; Goldman M; van der Ploeg AT; Bratkovic D; Kuchipudi S; Mozaffar T; Kishnani PS;
Lancet Neurol; 2021 Dec; 20(12):1027-1037. PubMed ID: 34800400
[TBL] [Abstract][Full Text] [Related]
26. Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease.
Kanters TA; van der Ploeg AT; Kruijshaar ME; Rizopoulos D; Redekop WK; Rutten-van Mӧlken MPMH; Hakkaart-van Roijen L
Orphanet J Rare Dis; 2017 Dec; 12(1):179. PubMed ID: 29237491
[TBL] [Abstract][Full Text] [Related]
27. Spanish Pompe registry: Baseline characteristics of first 49 patients with adult onset of Pompe disease.
Alonso-Pérez J; Segovia S; Domínguez-González C; Olivé M; Mendoza Grimón MD; Fernández-Torrón R; López de Munain A; Muñoz-Blanco JL; Ramos-Fransi A; Almendrote M; Illa I; Díaz-Manera J
Med Clin (Barc); 2020 Feb; 154(3):80-85. PubMed ID: 31253477
[TBL] [Abstract][Full Text] [Related]
28. 24-months results in two adults with Pompe disease on enzyme replacement therapy.
Vielhaber S; Brejova A; Debska-Vielhaber G; Kaufmann J; Feistner H; Schoenfeld MA; Awiszus F
Clin Neurol Neurosurg; 2011 Jun; 113(5):350-7. PubMed ID: 21477922
[TBL] [Abstract][Full Text] [Related]
29. Effect of long term enzyme replacement therapy in late onset Pompe disease: A single-centre experience.
Papadimas GK; Anagnostopoulos C; Xirou S; Michelakakis H; Terzis G; Mavridou I; Kararizou E; Papadopoulos C
Neuromuscul Disord; 2021 Feb; 31(2):91-100. PubMed ID: 33451932
[TBL] [Abstract][Full Text] [Related]
30. Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease.
Dimachkie MM; Barohn RJ; Byrne B; Goker-Alpan O; Kishnani PS; Ladha S; Laforêt P; Mengel KE; Peña LDM; Sacconi S; Straub V; Trivedi J; Van Damme P; van der Ploeg AT; Vissing J; Young P; Haack KA; Foster M; Gilbert JM; Miossec P; Vitse O; Zhou T; Schoser B;
Neurology; 2022 Aug; 99(5):e536-e548. PubMed ID: 35618441
[TBL] [Abstract][Full Text] [Related]
31. Distal muscle weakness is a common and early feature in long-term enzyme-treated classic infantile Pompe patients.
van den Dorpel JJA; Poelman E; Harlaar L; van Kooten HA; van der Giessen LJ; van Doorn PA; van der Ploeg AT; van den Hout JMP; van der Beek NAME
Orphanet J Rare Dis; 2020 Sep; 15(1):247. PubMed ID: 32928284
[TBL] [Abstract][Full Text] [Related]
32. Cardiopulmonary exercise test to quantify enzyme replacement response in pediatric Pompe disease.
Bar-Yoseph R; Mandel H; Mainzer G; Gur M; Tal G; Shalloufeh G; Bentur L
Pediatr Pulmonol; 2018 Mar; 53(3):366-373. PubMed ID: 29356433
[TBL] [Abstract][Full Text] [Related]
33. Description of clinical and genetic features of 122 patients included in the Spanish Pompe registry.
Martinez-Marin RJ; Reyes-Leiva D; Nascimento A; Muelas N; Dominguez-González C; Paradas C; Olivé M; García-Romero M; Pascual-Pascual SI; Grau JM; Barba-Romero MA; Gomez-Caravaca MT; de Las Heras J; Casquero P; Mendoza MD; de León JC; Gutierrez A; Morís G; Blanco-Lago R; Ramos-Fransi A; Pintós G; García-Antelo MJ; Rabasa M; Morgado Y; Usón M; Miralles FJ; Bárcena-Llona JE; Gómez-Belda AB; Pedraza-Hueso MI; Hortelano M; Colomé A; Garcia-Martin G; Lopez de Munain A; Jericó I; Galán-Dávila L; Pardo J; Salgueiro-Origlia G; Alonso-Pérez J; Pla-Junca F; Schiava M; Segovia-Simón S; Díaz-Manera J
Neuromuscul Disord; 2024 Jan; 34():1-8. PubMed ID: 38087756
[TBL] [Abstract][Full Text] [Related]
34. 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; 15():1336599. PubMed ID: 38715621
[TBL] [Abstract][Full Text] [Related]
35. Pompe disease: design, methodology, and early findings from the Pompe Registry.
Byrne BJ; Kishnani PS; Case LE; Merlini L; Müller-Felber W; Prasad S; van der Ploeg A
Mol Genet Metab; 2011 May; 103(1):1-11. PubMed ID: 21439876
[TBL] [Abstract][Full Text] [Related]
36. 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
[TBL] [Abstract][Full Text] [Related]
37. Effectiveness of enzyme replacement therapy in adults with late-onset Pompe disease: results from the NCS-LSD cohort study.
Anderson LJ; Henley W; Wyatt KM; Nikolaou V; Waldek S; Hughes DA; Lachmann RH; Logan S
J Inherit Metab Dis; 2014 Nov; 37(6):945-52. PubMed ID: 24906254
[TBL] [Abstract][Full Text] [Related]
38. Early-onset of symptoms and clinical course of Pompe disease associated with the c.-32-13 T > G variant.
Herbert M; Case LE; Rairikar M; Cope H; Bailey L; Austin SL; Kishnani PS
Mol Genet Metab; 2019 Feb; 126(2):106-116. PubMed ID: 30655185
[TBL] [Abstract][Full Text] [Related]
39. Long-term effects of enzyme replacement therapy in an elderly cohort of late-onset Pompe disease.
Winkler M; von Landenberg C; Kuchenbecker K; Reimann J; Kornblum C
Neuromuscul Disord; 2022 Mar; 32(3):195-205. PubMed ID: 35120758
[TBL] [Abstract][Full Text] [Related]
40. Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy.
Güngör D; de Vries JM; Hop WC; Reuser AJ; van Doorn PA; van der Ploeg AT; Hagemans ML
Orphanet J Rare Dis; 2011 Jun; 6():34. PubMed ID: 21631931
[TBL] [Abstract][Full Text] [Related]
[Previous] [Next] [New Search]