303 related articles for article (PubMed ID: 22002767)
1. Enzyme replacement therapy for Pompe disease.
Angelini C; Semplicini C
Curr Neurol Neurosci Rep; 2012 Feb; 12(1):70-5. PubMed ID: 22002767
[TBL] [Abstract][Full Text] [Related]
2. Pompe Disease: New Developments in an Old Lysosomal Storage Disorder.
Meena NK; Raben N
Biomolecules; 2020 Sep; 10(9):. PubMed ID: 32962155
[TBL] [Abstract][Full Text] [Related]
3. Aerobic training as an adjunctive therapy to enzyme replacement in Pompe disease.
Nilsson MI; Samjoo IA; Hettinga BP; Koeberl DD; Zhang H; Hawke TJ; Nissar AA; Ali T; Brandt L; Ansari MU; Hazari H; Patel N; Amon J; Tarnopolsky MA
Mol Genet Metab; 2012 Nov; 107(3):469-79. PubMed ID: 23041258
[TBL] [Abstract][Full Text] [Related]
4. Evaluating avalglucosidase alfa for the management of late-onset Pompe disease.
Angelini C
Expert Rev Neurother; 2024 Mar; 24(3):259-266. PubMed ID: 38261315
[TBL] [Abstract][Full Text] [Related]
5. Restoration of muscle functionality by genetic suppression of glycogen synthesis in a murine model of Pompe disease.
Douillard-Guilloux G; Raben N; Takikita S; Ferry A; Vignaud A; Guillet-Deniau I; Favier M; Thurberg BL; Roach PJ; Caillaud C; Richard E
Hum Mol Genet; 2010 Feb; 19(4):684-96. PubMed ID: 19959526
[TBL] [Abstract][Full Text] [Related]
6. Gene Therapy for Pompe Disease: The Time is now.
Colella P; Mingozzi F
Hum Gene Ther; 2019 Oct; 30(10):1245-1262. PubMed ID: 31298581
[TBL] [Abstract][Full Text] [Related]
7. Infantile Pompe disease on ERT: update on clinical presentation, musculoskeletal management, and exercise considerations.
Case LE; Beckemeyer AA; Kishnani PS
Am J Med Genet C Semin Med Genet; 2012 Feb; 160C(1):69-79. PubMed ID: 22252989
[TBL] [Abstract][Full Text] [Related]
8. Suppression of autophagy permits successful enzyme replacement therapy in a lysosomal storage disorder--murine Pompe disease.
Raben N; Schreiner C; Baum R; Takikita S; Xu S; Xie T; Myerowitz R; Komatsu M; Van der Meulen JH; Nagaraju K; Ralston E; Plotz PH
Autophagy; 2010 Nov; 6(8):1078-89. PubMed ID: 20861693
[TBL] [Abstract][Full Text] [Related]
9. New insights into therapeutic options for Pompe disease.
Richard E; Douillard-Guilloux G; Caillaud C
IUBMB Life; 2011 Nov; 63(11):979-86. PubMed ID: 22002928
[TBL] [Abstract][Full Text] [Related]
10. [The efficacy of enzyme replacement therapy in pompe disease and secondary pathology in pompe muscle "excessive autophagy" which is relevant to the therapy resistance].
Fukuda T
No To Hattatsu; 2010 Mar; 42(2):114-6. PubMed ID: 23858594
[No Abstract] [Full Text] [Related]
11. Autophagy in skeletal muscle: implications for Pompe disease.
Shea L; Raben N
Int J Clin Pharmacol Ther; 2009; 47 Suppl 1(Suppl 1):S42-7. PubMed ID: 20040311
[TBL] [Abstract][Full Text] [Related]
12. The new era of Pompe disease: advances in the detection, understanding of the phenotypic spectrum, pathophysiology, and management.
Kishnani PS; Beckemeyer AA; Mendelsohn NJ
Am J Med Genet C Semin Med Genet; 2012 Feb; 160C(1):1-7. PubMed ID: 22253049
[TBL] [Abstract][Full Text] [Related]
13. A cross-sectional single-centre study on the spectrum of Pompe disease, German patients: molecular analysis of the GAA gene, manifestation and genotype-phenotype correlations.
Herzog A; Hartung R; Reuser AJ; Hermanns P; Runz H; Karabul N; Gökce S; Pohlenz J; Kampmann C; Lampe C; Beck M; Mengel E
Orphanet J Rare Dis; 2012 Jun; 7():35. PubMed ID: 22676651
[TBL] [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; 34(10):834-9. PubMed ID: 22521436
[TBL] [Abstract][Full Text] [Related]
15. Enzyme replacement therapy for infantile Pompe disease during the critical period and identification of a novel mutation.
But WM; Lee SH; Chan AO; Lau GT
Hong Kong Med J; 2009 Dec; 15(6):474-7. PubMed ID: 19966354
[TBL] [Abstract][Full Text] [Related]
16. Long-term observational, non-randomized study of enzyme replacement therapy in late-onset glycogenosis type II.
Bembi B; Pisa FE; Confalonieri M; Ciana G; Fiumara A; Parini R; Rigoldi M; Moglia A; Costa A; Carlucci A; Danesino C; Pittis MG; Dardis A; Ravaglia S
J Inherit Metab Dis; 2010 Dec; 33(6):727-35. PubMed ID: 20838899
[TBL] [Abstract][Full Text] [Related]
17. Advances in diagnosis and management of Pompe disease.
Davison JE
J Mother Child; 2020 Oct; 24(2):3-8. PubMed ID: 33554498
[TBL] [Abstract][Full Text] [Related]
18. [Pompe's disease. Part II. Treatment strategies and enzyme replacement].
Illés Z; Várdi Visy K
Ideggyogy Sz; 2009 Sep; 62(9-10):299-307. PubMed ID: 19835271
[TBL] [Abstract][Full Text] [Related]
19. 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]
20. Pompe disease (glycogen storage disease type II): clinical features and enzyme replacement therapy.
van der Beek NA; Hagemans ML; van der Ploeg AT; Reuser AJ; van Doorn PA
Acta Neurol Belg; 2006 Jun; 106(2):82-6. PubMed ID: 16898258
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
