World Orphan Drug Congress USA 2016
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Interview with Mark Rothera, PTC Therapeutics

Interview with Mark Rothera, PTC Therapeutics

Download this eBook now to find out more about:

  • Game-changer Translarna for over 2,000 rare diseases with a nonsense mutation. What makes Translarna unique?
  • What’s next for Translarna after the latest Phase 3 clinical trials
  • Reactions to Biomarin’s previously GSK/Prosensa’s orphan drug disappointing Ph 3 study results and the pending Sarepta results
  • How to choose which to prioritize from among the 2,000 rare diseases with a nonsense mutation
  •  The most challenging part in getting approval in Europe
  • How early collaboration with regulators should be
  • What processes or steps helped along this regulatory collaboration and approval
  • Market access
  • At what point would PTC sell

And of course a sneak preview of Mark's keynote session at the World Orphan Drug Congress USA next April.
 

Get your copy here >

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