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Volumn 20, Issue 12, 2010, Pages 773-774

Where do we stand in enzyme replacement therapy in Pompe's disease?

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA GLUCOSIDASE; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE;

EID: 78649360686     PISSN: 09608966     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.nmd.2010.09.011     Document Type: Editorial
Times cited : (25)

References (10)
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    • Van Capelle C.I., Van der Beek N.A.M.E., Hagemans M.L.C., et al. Effect of enzyme therapy in juvenile patients with Pompe disease: a three-year open label study. Neuromuscul Disord 2010, 20:775-782.
    • (2010) Neuromuscul Disord , vol.20 , pp. 775-782
    • Van Capelle, C.I.1    Van der Beek, N.A.M.E.2    Hagemans, M.L.C.3
  • 3
    • 77950963839 scopus 로고    scopus 로고
    • A randomized study of alglucosidase Alfa in late onset Pompe's disease
    • Van der Ploeg A.T., Clemens P.R., Corzo D., et al. A randomized study of alglucosidase Alfa in late onset Pompe's disease. N Engl J Med 2010, 362:1396-1406.
    • (2010) N Engl J Med , vol.362 , pp. 1396-1406
    • Van der Ploeg, A.T.1    Clemens, P.R.2    Corzo, D.3
  • 4
    • 33846033132 scopus 로고    scopus 로고
    • Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease
    • Kishnani P.S., Corzo D., Nicolino M., et al. Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease. Neurology 2007, 68:99-109.
    • (2007) Neurology , vol.68 , pp. 99-109
    • Kishnani, P.S.1    Corzo, D.2    Nicolino, M.3
  • 5
    • 70350448214 scopus 로고    scopus 로고
    • Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease
    • Kishnani P.S., Corzo D., Leslie N.D., et al. Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease. Pediatr Res 2009 Sep, 66(3):329-335.
    • (2009) Pediatr Res , vol.66 , Issue.3 , pp. 329-335
    • Kishnani, P.S.1    Corzo, D.2    Leslie, N.D.3
  • 6
    • 33644994280 scopus 로고    scopus 로고
    • Course of disability and respiratory function in untreated late-onset Pompe disease
    • Hagemans M.L., Hop W.J., Van Doorn P.A., Reuser A.J., Van der Ploeg A.T. Course of disability and respiratory function in untreated late-onset Pompe disease. Neurology 2006, 66:581-583.
    • (2006) Neurology , vol.66 , pp. 581-583
    • Hagemans, M.L.1    Hop, W.J.2    Van Doorn, P.A.3    Reuser, A.J.4    Van der Ploeg, A.T.5
  • 7
    • 53549095724 scopus 로고    scopus 로고
    • Clinical features of late-onset Pompe disease: a prospective cohort study
    • Wokke J.H., Escolar D.M., Pestronk A., et al. Clinical features of late-onset Pompe disease: a prospective cohort study. Muscle Nerve 2009, 38:1236-1245.
    • (2009) Muscle Nerve , vol.38 , pp. 1236-1245
    • Wokke, J.H.1    Escolar, D.M.2    Pestronk, A.3
  • 8
    • 59149086832 scopus 로고    scopus 로고
    • Rate of disease progression during long-term follow-up of patients with late-onset Pompe disease
    • Van der Beek N.A., Hagemans M.L., Reuser A.J., et al. Rate of disease progression during long-term follow-up of patients with late-onset Pompe disease. Neuromuscul Disord 2009, 19:113-117.
    • (2009) Neuromuscul Disord , vol.19 , pp. 113-117
    • Van der Beek, N.A.1    Hagemans, M.L.2    Reuser, A.J.3
  • 9
    • 45449088878 scopus 로고    scopus 로고
    • Eight years experience with enzyme replacement therapy in two children and one adult with Pompe disease
    • Van Capelle C.I., Winkel L.P., Hagemans M.L., et al. Eight years experience with enzyme replacement therapy in two children and one adult with Pompe disease. Neuromuscul Disord 2008, 18:447-45222.
    • (2008) Neuromuscul Disord , vol.18 , pp. 447-45222
    • Van Capelle, C.I.1    Winkel, L.P.2    Hagemans, M.L.3
  • 10
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    • 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., et al. 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. J Neurol 2009, 257:91-97.
    • (2009) J Neurol , vol.257 , pp. 91-97
    • Strothotte, S.1    Strigl-Pill, N.2    Grunert, B.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.