Titre : | International Pompe Conference 2003 - Vol 3-4 |
Auteurs : | International Pompe Conference (1-2 Novembre 2003; Heidelberg, Germany) ; Shapira S ; House T ; Schaller TH ; Amalfitano A ; Voit TH ; Bajbouj M ; Ollington F ; van der Ploeg A ; Kishnani P ; Wijnem I |
Type de document : | Multimédia |
Editeur : | Buren : Selbsthilfegruppe Glykogenose Deutschlande e.V, 2003 |
Langues: | Anglais |
Mots-clés : | colloque ; conférence d'experts ; enfant ; essai clinique ; gène GAA ; maladie de Pompe ; parent ; thérapie génique |
Résumé : | Vol-3 - Workshop Discussion of Patient and -Médical Experiences with Physical Therapy -Guest presentation : Enzyme Replacement Therapy - Experience with enzyme replacement therapy in patients with mocopolysacchari- dose type 1 -Enzyme Replacement Therapy in Pompe's Disease : Experiences and Perspectives - Rotterdam experience with enzyme replacement therapy for Pompe's disease - Enzyme replacement therapy with recombinant human acid alpha glucosidase in infantile Pompe disease : Duke experience -Experiences of parents of a Pompe infant under treatment Vol -4 -Experiences of a juvenile Pompe patient under treatment (video presentation) -Gene Therapy - Potential of gene therapy for GSD-II - Discussion of Session C4 |