Nouveaux traitements dans la mucoviscidose
Nouveaux traitements dans la mucoviscidose
Nouveaux traitements dans la mucoviscidose
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Ataluren - Essai phase 2aScreeningRandomization2 weeks2 weeksPTC124Dose Level(mg/kg/dose)4, 4, 8 TID10, 10, 20 TIDCharacteristicn=30Genotype (PTC)n=30Age, median [range], yearsPulmonary function, %-predicted b 12 [6-18]G542X (UGA)W1282X (UGA)144FEV 190 [40-133]Q493X (UAG)3FVCPathological bacterial/fungal colonization, nPseudomonas aeruginosaOther (eg,Staphylococcus, Aspergillus)99 [52-131]30926R553X (UGA)E1104X (UGA)R1162X (UGA)W846X (UGA)W882X (UAG)22211Q1313X (UAA)1Sermet-Gaudelus et al. AJRCCM 2010
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