171 related articles for article (PubMed ID: 37419682)
1. 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]
2. 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]
3. The French Pompe registry. Baseline characteristics of a cohort of 126 patients with adult Pompe disease.
Laforêt P; Laloui K; Granger B; Hamroun D; Taouagh N; Hogrel JY; Orlikowski D; Bouhour F; Lacour A; Salort-Campana E; Penisson-Besnier I; Sacconi S; Zagnoli F; Chapon F; Eymard B; Desnuelle C; Pouget J;
Rev Neurol (Paris); 2013; 169(8-9):595-602. PubMed ID: 24008051
[TBL] [Abstract][Full Text] [Related]
4. 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]
5. Real-life effectiveness 1 year after switching to avalglucosidase alfa in late-onset Pompe disease patients worsening on alglucosidase alfa therapy: A French cohort study.
Tard C; Bouhour F; Michaud M; Beltran S; Fournier M; Demurger F; Lagrange E; Nollet S; Sacconi S; Noury JB; Magot A; Cintas P; Renard D; Deibener-Kaminsky J; Lefeuvre C; Davion JB; Salort-Campana E; Arrassi A; Taouagh N; Spinazzi M; ; Attarian S; Laforêt P
Eur J Neurol; 2024 Jul; 31(7):e16292. PubMed ID: 38587143
[TBL] [Abstract][Full Text] [Related]
6. 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]
7. 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; 25(4):321-32. PubMed ID: 25617983
[TBL] [Abstract][Full Text] [Related]
8. 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]
9. Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial.
Diaz-Manera J; Kishnani PS; Kushlaf H; Ladha S; Mozaffar T; Straub V; Toscano A; van der Ploeg AT; Berger KI; Clemens PR; Chien YH; Day JW; Illarioshkin S; Roberts M; Attarian S; Borges JL; Bouhour F; Choi YC; Erdem-Ozdamar S; Goker-Alpan O; Kostera-Pruszczyk A; Haack KA; Hug C; Huynh-Ba O; Johnson J; Thibault N; Zhou T; Dimachkie MM; Schoser B;
Lancet Neurol; 2021 Dec; 20(12):1012-1026. PubMed ID: 34800399
[TBL] [Abstract][Full Text] [Related]
10. 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]
11. 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]
12. 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]
13. Motor and respiratory decline in patients with late onset Pompe disease after cessation of enzyme replacement therapy during COVID-19 pandemic.
Tard C; Salort-Campana E; Michaud M; Spinazzi M; Nadaj Pakleza A; Durr H; Bouhour F; Lefeuvre C; Thomas R; Arrassi A; Taouagh N; ; Solé G; Laforêt P
Eur J Neurol; 2022 Apr; 29(4):1181-1186. PubMed ID: 34927321
[TBL] [Abstract][Full Text] [Related]
14. Enzyme replacement therapy for late-onset Pompe disease.
Dalmia S; Sharma R; Ramaswami U; Hughes D; Jahnke N; Cole D; Smith S; Remmington T
Cochrane Database Syst Rev; 2023 Dec; 12(12):CD012993. PubMed ID: 38084761
[TBL] [Abstract][Full Text] [Related]
15. 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;
Lancet Child Adolesc Health; 2022 Jan; 6(1):28-37. PubMed ID: 34822769
[TBL] [Abstract][Full Text] [Related]
16. 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]
17. Hypersensitivity infusion-associated reactions induced by enzyme replacement therapy in a cohort of patients with late-onset Pompe disease: An experience from the French Pompe Registry.
Lessard LER; Tard C; Salort-Campana E; Sacconi S; Béhin A; Bassez G; Orlikowski D; Merle P; Nollet S; Gallay L; Bérard F; Robinson P; Bouhour F; Laforêt P
Mol Genet Metab; 2023 Jul; 139(3):107611. PubMed ID: 37285781
[TBL] [Abstract][Full Text] [Related]
18. 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; 257(1):91-7. PubMed ID: 19649685
[TBL] [Abstract][Full Text] [Related]
19. Enzyme replacement therapy for infantile-onset Pompe disease.
Chen M; Zhang L; Quan S
Cochrane Database Syst Rev; 2017 Nov; 11(11):CD011539. PubMed ID: 29155436
[TBL] [Abstract][Full Text] [Related]
20. Survival and long-term outcomes in late-onset Pompe disease following alglucosidase alfa treatment: a systematic review and meta-analysis.
Schoser B; Stewart A; Kanters S; Hamed A; Jansen J; Chan K; Karamouzian M; Toscano A
J Neurol; 2017 Apr; 264(4):621-630. PubMed ID: 27372449
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]