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Washington Court Hotel

25 sep 2013 8:00 a.m. - 27 sep 2013 5:00 p.m.

525 New Jersey Avenue NW, Washington, DC 20001

DIA/FDA Oligonucleotide-based Therapeutics Conference

Session Chair(s)

Lois  Freed, PHD

Lois Freed, PHD

Director, Division of Pharmacology/Toxicology-Neuroscience (DPT-N), CDER

FDA, United States

Stephen B. Shrewsbury, MD, FFPM

Stephen B. Shrewsbury, MD, FFPM

Advisory Board

LifeSplice Pharma, Canada

Speaker(s)

Richard Stephen Geary, PHD

Antisense Therapies for Neurodegenerative Diseases

Richard Stephen Geary, PHD

Ionis Pharmaceuticals, Inc., United States

Senior Vice President, Drug Development

Jerry R Mendell, MD

Update on Sarepta’s AVI-4658 in Exon 51 Skipping Amenable Duchenne Muscular Dystrophy Boys

Jerry R Mendell, MD

Nationwide Children's Hospital, United States

Professor of Pediatrics, Neurology, Pathology & Physiology

Craig  McDonald, MD

Update on GSK’s Drisapersen in Exon 51 Skipping Amenable DMD

Craig McDonald, MD

UC Davis Children's Hospital, United States

Giles  Campion, MD, PHD

Update on Prosensa’s other Splice Switching Oligomers (PRO044) in DMD

Giles Campion, MD, PHD

BioMarin Nederland BV, Netherlands

Chief Medical Officer

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