Effect of enzyme replacement therapy with alglucosidase alfa (Myozyme®) in 12 patients with advanced late-onset Pompe disease

Constantinos Papadopoulos; David Orlikowski; Hélène Prigent; Arnaud Lacour; Céline Tard; Alain Furby; Julien Praline; Guilhem Solé; Jean-Yves Hogrel; Marie De Antonio; Claudio Semplicini; Joelle Deibener-Kaminsky; Pierre Kaminsky; Bruno Eymard; Nadjib Taouagh; Barbara Perniconi; Dalil Hamroun; Pascal Laforêt; G. Bassez; A.-L. Bedat-Millet; A. Behin; B. Eymard; S. Leonard-Louis; T. Stojkovic; A. Canal; V. Decostre; F. Bouhour; F. Boyer; C. Caillaud; Y. Castaing; F. Chapon; P. Cintas; I. Durieu; A. Echaniz-Laguna; L. Feasson; X. Ferrer; R. Froissart; M. Piraud; D. Germain; K. Benistan; N. Guffon-Fouilhoux; H. Journel; P. Labauge; A. Levy; A. Magot; Y. Péréon; M.-C. Minot-Myhié; A. Nadaj-Pakleza; C. Nathier; N. Pellegrini; P. Petiot; F. Lofaso; A. Dutry; D. Renard; S. Sacconi; C. Desnuelle; E. Salort-Campana; J. Pouget; V. Tiffreau; D. Vincent; F. Zagnoli

doi:10.1016/j.ymgme.2017.06.007

Effect of enzyme replacement therapy with alglucosidase alfa (Myozyme®) in 12 patients with advanced late-onset Pompe disease

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2017.06.007 ◽

2017 ◽

Vol 122 (1-2) ◽

pp. 80-85 ◽

Cited By ~ 14

Author(s):

Constantinos Papadopoulos ◽

David Orlikowski ◽

Hélène Prigent ◽

Arnaud Lacour ◽

Céline Tard ◽

...

Keyword(s):

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Enzyme Replacement ◽

Alglucosidase Alfa

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A 4-year follow-up study of 24 patients with late onset Pompe disease treated with alglucosidase alfa enzyme replacement therapy at a single centre

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2014.12.252 ◽

2015 ◽

Vol 114 (2) ◽

pp. S111

Author(s):

Karolina M. Stepien ◽

Jane Whitby ◽

Mark Roberts ◽

Reena Sharma

Keyword(s):

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Single Centre ◽

Enzyme Replacement ◽

Follow Up Study ◽

Alglucosidase Alfa

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Phase 1 safety and pharmacokinetics of the novel enzyme replacement therapy neoGAA in treatment-naïve and alglucosidase alfa-treated late-onset Pompe disease patients

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2015.12.476 ◽

2016 ◽

Vol 117 (2) ◽

pp. S117-S118

Author(s):

Shafeeq Ladha ◽

Pascal Laforet ◽

Eugen Mengel ◽

Alan Pestronk ◽

Jean Pouget ◽

...

Keyword(s):

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Phase 1 ◽

The Novel ◽

Enzyme Replacement ◽

Alglucosidase Alfa ◽

Treatment Naïve

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Phase 1 exploratory efficacy of the novel enzyme replacement therapy neoGAA in treatment-naïve and alglucosidase alfa-treated late-onset Pompe disease patients

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2015.12.396 ◽

2016 ◽

Vol 117 (2) ◽

pp. S92

Author(s):

Loren Pena ◽

Richard Baron ◽

Barry Byrne ◽

Claude Desnuelle ◽

Ozlem Goker-Alpan ◽

...

Keyword(s):

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Phase 1 ◽

The Novel ◽

Enzyme Replacement ◽

Alglucosidase Alfa ◽

Treatment Naïve

Download Full-text