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Author The French Pompe Registry Study Group |
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Article
Congrès international de myologie 2008 (International Congress of Myology 2008; 26-30 mai 2008; Marseille, France) ; De Castro D ; Laloui K ; Doppler V ; Payan C ; Hogrel JY ; Ollivier G ; Laforet P ; The French Pompe Registry Study Group | 2008Introduction: Recombinant human alpha al-glucosidase (rhGAA; Myozyme® Genzyme), an enzyme replacement therapy (ERT) for Pompe disease (PD; acid maltase deficiency, glycogenosis type II), was first used in France for late-onset patients in May 20[...]