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Amicus CEO Represents BIO on Rare Diseases at U.S. Senate Committee on Health, Education, Labor and Pensions Bipartisan Hearing
Date:7/21/2010

duct approval, either for full approval or accelerated approval purposes;
  • Improving standards for demonstrating safety and efficacy of rare disease products;
  • Improving communications processes for rare disease stakeholders;
  • Extending the Qualifying Therapeutic Discovery Project Tax Credit; and
  • Fully funding the Cures Acceleration Network (CAN).

  • John F. Crowley commented in his testimony, "We have come very far in the last quarter of a century but we have much further to go. The change brought about by the Orphan Drug Act improved millions of lives in this country and abroad, helped launch an industry and established the global rare disease advocacy movement. It does not come easily for every family that struggles with illnesses and then receives a life-altering diagnosis of a rare disease with no treatment or cure. However, each of us committed to orphan drug development, including the FDA and those responsible for seeing the Agency is appropriately funded, owes those families a more-than-fighting chance that their medical needs will be met."

    "Through his compelling personal story and his professional passion, John Crowley truly understands the importance of the Orphan Drug Act and the need for additional incentives to spur the development of breakthrough treatments for rare and orphan diseases," said BIO President and CEO Jim Greenwood. "The recommendations John made in his testimony today reflect the concerns and interests of our member companies who are actively researching the next generation of therapies and the patients and their families who so desperately need them."

    About Amicus Therapeutics

    Amicus Therapeutics is a biopharmaceutical company focused on developing treatments for rare d
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