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Vídeo 1: Parent Perspective: Impact of DMD.
Vídeo 2: Natural history of DMD and standards of care.
Vídeo 3: Genetics of DMD and the exon skipping approach.
Vídeo 4: 2-‘O-methyl phosporothioate chemistry.
Vídeo 5: Phosphorodiamidate morpholino oligomer chemistry.
Vídeo 6: Overview of next generation chemistries.
Vídeo 7: Regulatory models for marketing authorization.
Vídeo 8: Discussion.
Vídeo 9: Overview of functional outcome measures used in DMD clinical trials.
Vídeo 10: Moleculars biomarkers for DMD.
Vídeo 11: Magnetic resonance imaging (MRI) for DMD.
Vídeo 12: Patient perspective to clinical benefit and outcome measures.
Vídeo 13: Discussion.
Vídeo 14: Patient perspective on risk-benefit analysis.
Vídeo 15: Regulatory perspective on risk-benefit analysis.
Vídeo 16: Discussion and end of meeting.