Pharmacopée actuelle

Nathalie Goemans
Durée : 2004000
00
Introduction
Drugs development in DMD : where are we today ?
Therapeutic options and strategies
Overview
PTC124/ Translama (Atularen) - Ribosomal readthrough molecule
Ataluren Conformatory Trial (ACT DMD) : Results
Clinical trials exon skipping 51
Different stage of disease at inclusion
Long-term treatment effect - By Parent Study Analysis
Adverse reactions
Exit Drisapersen
Exondys 51 (Sarepta/Eteplirsen)
Clinical efficacy (6MWT) comparison with Natural History data
Eteplirsen > Exondys 51 (Sarepta)
Tadalafil, a Phosphodierase Type 5 Inhibitor
Idebenone (Raxone)
Phases I en II Studies
Conclusions
Questions -réponses
  • Introduction (0)
  • Drugs development in DMD : where are we today ? (49000)
  • Therapeutic options and strategies (114000)
  • Overview (160000)
  • PTC124/ Translama (Atularen) - Ribosomal readthrough molecule (242000)
  • Ataluren Conformatory Trial (ACT DMD) : Results (404000)
  • Clinical trials exon skipping 51 (498000)
  • Different stage of disease at inclusion (616000)
  • Long-term treatment effect - By Parent Study Analysis (653000)
  • Adverse reactions (775000)
  • Exit Drisapersen (820000)
  • Exondys 51 (Sarepta/Eteplirsen) (852000)
  • Clinical efficacy (6MWT) comparison with Natural History data (923000)
  • Eteplirsen > Exondys 51 (Sarepta) (962000)
  • Tadalafil, a Phosphodierase Type 5 Inhibitor (1047000)
  • Idebenone (Raxone) (1129000)
  • Phases I en II Studies (1352000)
  • Conclusions (1416000)
  • Questions -réponses (1518000)
Nathalie Goemans

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