Titre : | Disease progression in a pre-symptomatically treated patient with juvenile-onset Pompe disease - need for an earlier treatment? |
Revue : | European journal of neurology, 25, 10 |
Auteurs : | Chien YH, Auteur ; Lee NC ; Hwu WL ; Fang JY |
Type de document : | Article |
Année de publication : | 10/2018 |
Langues: | Anglais |
Mots-clés : | recommandation |
Mots-clés: | Pompe disease;acid α-glucosidase;enzyme replacement therapy;magnetic resonance imaging;pre-symptomatic hyperCKaemia; |
Résumé : |
Comment on:
European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10-year experience. [Eur J Neurol. 2017] |
Pubmed / DOI : | Pubmed : 30192061 / DOI : 10.1111/ene.13730 |
N° Profil MNM : | 2018091 |
En ligne : | http://www.ncbi.nlm.nih.gov/pubmed/30192061 |