180 related articles for article (PubMed ID: 34834457)
1. Individualized Assessment of Exercise Capacity in Response to Acute and Long-Term Enzyme Replacement Therapy in Pediatric Pompe Disease.
Bar-Yoseph R; Tal G; Dumin E; Hanna M; Mainzer G; Zucker-Toledano M; Shallufi G; Jahshan M; Mandel H; Bentur L
J Pers Med; 2021 Oct; 11(11):. PubMed ID: 34834457
[TBL] [Abstract][Full Text] [Related]
2. 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]
3. Role of the cardio-pulmonary exercise test and six-minute walking test in the evaluation of exercise performance in patients with late-onset Pompe disease.
Crescimanno G; Modica R; Lo Mauro R; Musumeci O; Toscano A; Marrone O
Neuromuscul Disord; 2015 Jul; 25(7):542-7. PubMed ID: 25908581
[TBL] [Abstract][Full Text] [Related]
4. Earlier and higher dosing of alglucosidase alfa improve outcomes in patients with infantile-onset Pompe disease: Evidence from real-world experiences.
Chien YH; Tsai WH; Chang CL; Chiu PC; Chou YY; Tsai FJ; Wong SL; Lee NC; Hwu WL
Mol Genet Metab Rep; 2020 Jun; 23():100591. PubMed ID: 32373469
[TBL] [Abstract][Full Text] [Related]
5. The characterisation of pulmonary function in patients with mucopolysaccharidoses IVA: A longitudinal analysis.
Kenth JJ; Thompson G; Fullwood C; Wilkinson S; Jones S; Bruce IA
Mol Genet Metab Rep; 2019 Sep; 20():100487. PubMed ID: 31341787
[TBL] [Abstract][Full Text] [Related]
6. Albuterol as an adjunctive treatment to enzyme replacement therapy in infantile-onset Pompe disease.
Chien YH; Hwu WL; Lee NC; Tsai FJ; Koeberl DD; Tsai WH; Chiu PC; Chang CL
Mol Genet Metab Rep; 2017 Jun; 11():31-35. PubMed ID: 28480166
[TBL] [Abstract][Full Text] [Related]
7. Motor outcomes in patients with infantile and juvenile Pompe disease: Lessons from neurophysiological findings.
Brassier A; Pichard S; Schiff M; Bouchereau J; Bérat CM; Caillaud C; Pion A; Khraiche D; Fauroux B; Oualha M; Barnerias C; Desguerre I; Hully M; Maquet M; Deladrière E; de Lonlay P; Gitiaux C
Mol Genet Metab; 2023 Aug; 139(4):107650. PubMed ID: 37454519
[TBL] [Abstract][Full Text] [Related]
8. Divergent clinical outcomes of alpha-glucosidase enzyme replacement therapy in two siblings with infantile-onset Pompe disease treated in the symptomatic or pre-symptomatic state.
Matsuoka T; Miwa Y; Tajika M; Sawada M; Fujimaki K; Soga T; Tomita H; Uemura S; Nishino I; Fukuda T; Sugie H; Kosuga M; Okuyama T; Umeda Y
Mol Genet Metab Rep; 2016 Dec; 9():98-105. PubMed ID: 27896132
[TBL] [Abstract][Full Text] [Related]
9. 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]
10. Cardiopulmonary Exercise Testing Reflects Improved Exercise Capacity in Response to Treatment in Morquio A Patients: Results of a 52-Week Pilot Study of Two Different Doses of Elosulfase Alfa.
Berger KI; Burton BK; Lewis GD; Tarnopolsky M; Harmatz PR; Mitchell JJ; Muschol N; Jones SA; Sutton VR; Pastores GM; Lau H; Sparkes R; Shaywitz AJ
JIMD Rep; 2018; 42():9-17. PubMed ID: 29159458
[TBL] [Abstract][Full Text] [Related]
11. Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy.
Parini R; De Lorenzo P; Dardis A; Burlina A; Cassio A; Cavarzere P; Concolino D; Della Casa R; Deodato F; Donati MA; Fiumara A; Gasperini S; Menni F; Pagliardini V; Sacchini M; Spada M; Taurisano R; Valsecchi MG; Di Rocco M; Bembi B
Orphanet J Rare Dis; 2018 Feb; 13(1):32. PubMed ID: 29422078
[TBL] [Abstract][Full Text] [Related]
12. The influence of a polymorphism in the gene encoding angiotensin converting enzyme (ACE) on treatment outcomes in late-onset Pompe patients receiving alglucosidase alfa.
Baek RC; Palmer R; Pomponio RJ; Lu Y; Ma X; McVie-Wylie AJ
Mol Genet Metab Rep; 2016 Sep; 8():48-50. PubMed ID: 27489778
[TBL] [Abstract][Full Text] [Related]
13. 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]
14. 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]
15. 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]
16. Enzyme replacement therapy with alglucosidase alfa in Pompe disease: Clinical experience with rate escalation.
Desai AK; Walters CK; Cope HL; Kazi ZB; DeArmey SM; Kishnani PS
Mol Genet Metab; 2018 Feb; 123(2):92-96. PubMed ID: 29289479
[TBL] [Abstract][Full Text] [Related]
17. 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]
18. Long-term outcome and unmet needs in infantile-onset Pompe disease.
Hahn A; Schänzer A
Ann Transl Med; 2019 Jul; 7(13):283. PubMed ID: 31392195
[TBL] [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; 265(1):159-164. PubMed ID: 29181627
[TBL] [Abstract][Full Text] [Related]
20. 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]
[Next] [New Search]
