Long‐term benefit of enzyme replacement therapy with alglucosidase alfa in adults with Pompe disease: Prospective analysis from the French Pompe Registry

Despite a wide clinical spectrum, the adult form of Pompe disease is the most common one, and represents more than 90% of diagnosed patients in France. Since the marketing of enzyme replacement therapy (alglucosidase alfa, Myozyme), all reports to date in adults demonstrated an improvement of the wa...

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Veröffentlicht in:	Journal of inherited metabolic disease 2020-11, Vol.43 (6), p.1219-1231
Hauptverfasser:	Semplicini, Claudio, De Antonio, Marie, Taouagh, Nadjib, Béhin, Anthony, Bouhour, Françoise, Echaniz‐Laguna, Andoni, Magot, Armelle, Nadaj‐Pakleza, Aleksandra, Orlikowski, David, Sacconi, Sabrina, Salort‐Campana, Emmanuelle, Solé, Guilhem, Tard, Céline, Zagnoli, Fabien, Hogrel, Jean‐Yves, Hamroun, Dalil, Laforêt, Pascal
Format:	Artikel
Sprache:	eng
Schlagworte:	Adults alglucosidase alfa enzyme replacement therapy Enzymes Glycogen storage disease type II late onset Pompe disease Life Sciences Patients Registry Respiratory function Walking
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