With more than 350 million people affected by rare disease globally, it’s essential to turn the tide on these
often-overlooked diseases. However, collaboration and innovation are continuing to make an impact in rare disease research. Learn more from these articles with varying topics on rare disease research and regulation changes:

  • Orphazyme Steams Ahead With Registration in Niemann Pick, Despite Endpoint Misses
  • Rare but Real: How One Startup Is Willing Itself to Lift China Orphan Drug Market
  • EUSA Sylvant License Marks Toehold Into US Rare Disease Market
  • EU Consults on Revised Guide for Orphan Drug Designation

Read it ahead of Rare Disease Summit in Boston, June 13–14, 2019, where you can connect with
key stakeholders and propel curative progress, orphan product success and patient advocacy.


Rare Disease Article Pack

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