NDLI: Forme adulte de la maladie de Pompe : les leçons du Myozyme® - Utilisation clinique des traitements innovants, repositionnés ou hors AMM : le vécu du terrain (1)

Content Provider	Semantic Scholar
Author	Laforêt, Pascal
Copyright Year	2019
Abstract	L’alglucosidase alfa ou Myozyme® reste le premier medicament innovant disponible pour traiter une myopathie de l’adulte. Cette experience originelle est riche d’enseignements tant sur le plan de la recherche que de la pratique clinique. Methodologie des essais, mise en œuvre du traitement au sein d’un service hospitalier, contribution du registre des patients, dans tous ces domaines l’experience acquise peut etre capitalisee pour preparer l’arrivee d’autres innovations therapeutiques.
Starting Page	18
Ending Page	21
Page Count	4
File Format	PDF HTM / HTML
DOI	10.1051/medsci/2019021
Alternate Webpage(s)	http://www.myobase.org/doc_num.php?explnum_id=18186
Alternate Webpage(s)	https://doi.org/10.1051/medsci%2F2019021
Volume Number	35
Language	English
Access Restriction	Open
Content Type	Text
Resource Type	Article

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