369 related articles for article (PubMed ID: 31619483)
1. Large variation in effects during 10 years of enzyme therapy in adults with Pompe disease.
Harlaar L; Hogrel JY; Perniconi B; Kruijshaar ME; Rizopoulos D; Taouagh N; Canal A; Brusse E; van Doorn PA; van der Ploeg AT; Laforêt P; van der Beek NAME
Neurology; 2019 Nov; 93(19):e1756-e1767. PubMed ID: 31619483
[TBL] [Abstract][Full Text] [Related]
2. Long-term benefit of enzyme replacement therapy in Pompe disease: A 5-year prospective study.
Kuperus E; Kruijshaar ME; Wens SCA; de Vries JM; Favejee MM; van der Meijden JC; Rizopoulos D; Brusse E; van Doorn PA; van der Ploeg AT; van der Beek NAME
Neurology; 2017 Dec; 89(23):2365-2373. PubMed ID: 29117951
[TBL] [Abstract][Full Text] [Related]
3. 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;
J Inherit Metab Dis; 2020 Nov; 43(6):1219-1231. PubMed ID: 32515844
[TBL] [Abstract][Full Text] [Related]
4. Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study.
de Vries JM; van der Beek NA; Hop WC; Karstens FP; Wokke JH; de Visser M; van Engelen BG; Kuks JB; van der Kooi AJ; Notermans NC; Faber CG; Verschuuren JJ; Kruijshaar ME; Reuser AJ; van Doorn PA; van der Ploeg AT
Orphanet J Rare Dis; 2012 Sep; 7():73. PubMed ID: 23013746
[TBL] [Abstract][Full Text] [Related]
5. 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]
6. Long-term follow-up of 17 patients with childhood Pompe disease treated with enzyme replacement therapy.
van der Meijden JC; Kruijshaar ME; Harlaar L; Rizopoulos D; van der Beek NAME; van der Ploeg AT
J Inherit Metab Dis; 2018 Nov; 41(6):1205-1214. PubMed ID: 29556838
[TBL] [Abstract][Full Text] [Related]
7. 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;
Mol Genet Metab; 2017 Sep; 122(1-2):80-85. PubMed ID: 28648663
[TBL] [Abstract][Full Text] [Related]
8. 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
[TBL] [Abstract][Full Text] [Related]
9. Cessation and resuming of alglucosidase alfa in Pompe disease: a retrospective analysis.
Hundsberger T; Rösler KM; Findling O
J Neurol; 2014 Sep; 261(9):1684-90. PubMed ID: 24923245
[TBL] [Abstract][Full Text] [Related]
10. Clinical efficacy of the enzyme replacement therapy in patients with late-onset Pompe disease: a systematic review and a meta-analysis.
Sarah B; Giovanna B; Emanuela K; Nadi N; Josè V; Alberto P
J Neurol; 2022 Feb; 269(2):733-741. PubMed ID: 33851281
[TBL] [Abstract][Full Text] [Related]
11. 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]
12. Observational clinical study in juvenile-adult glycogenosis type 2 patients undergoing enzyme replacement therapy for up to 4 years.
Angelini C; Semplicini C; Ravaglia S; Bembi B; Servidei S; Pegoraro E; Moggio M; Filosto M; Sette E; Crescimanno G; Tonin P; Parini R; Morandi L; Marrosu G; Greco G; Musumeci O; Di Iorio G; Siciliano G; Donati MA; Carubbi F; Ermani M; Mongini T; Toscano A;
J Neurol; 2012 May; 259(5):952-8. PubMed ID: 22081099
[TBL] [Abstract][Full Text] [Related]
13. 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]
14. 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; 35(5):837-45. PubMed ID: 22290025
[TBL] [Abstract][Full Text] [Related]
15. 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]
16. 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]
17. Enzyme replacement therapy and fatigue in adults with Pompe disease.
Güngör D; de Vries JM; Brusse E; Kruijshaar ME; Hop WC; Murawska M; van den Berg LE; Reuser AJ; van Doorn PA; Hagemans ML; Plug I; van der Ploeg AT
Mol Genet Metab; 2013 Jun; 109(2):174-8. PubMed ID: 23603069
[TBL] [Abstract][Full Text] [Related]
18. 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
[TBL] [Abstract][Full Text] [Related]
19. Characteristics of Patients With Late-Onset Pompe Disease in France: Insights From the French Pompe Registry in 2022.
Lefeuvre C; De Antonio M; Bouhour F; Tard C; Salort-Campana E; Lagrange E; Behin A; Sole G; Noury JB; Sacconi S; Magot A; Nadaj-Pakleza A; Lacour A; Beltran S; Spinazzi M; Cintas P; Renard D; Michaud M; Bedat-Millet AL; Prigent H; Taouagh N; Arrassi A; Hamroun D; Attarian S; Laforêt P;
Neurology; 2023 Aug; 101(9):e966-e977. PubMed ID: 37419682
[TBL] [Abstract][Full Text] [Related]
20. The impact of interrupting enzyme replacement therapy in late-onset Pompe disease.
Wenninger S; Gutschmidt K; Wirner C; Einvag K; Montagnese F; Schoser B
J Neurol; 2021 Aug; 268(8):2943-2950. PubMed ID: 33625582
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
