Noticias sobre Distrofia Muscular de Duchenne y Becker

Vídeos online del Workshop “Antisense oligonucleotide-mediated exon skipping therapy development for Duchenne muscular dystrophy (DMD)” – 29 April 2015.

Hacer click sobre las imágenes para acceder a los vídeos del Workshop.

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.


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