John Crowley on the 1-Year Anniversary of a Rare First Address

John Crowley

Last year, at President Donald Trump's First Address to a Joint Session of Congress, he made mention of one particular face in the crowd: Megan Crowley, a 20-year-old sophomore student at University of Notre Dame who was diagnosed with Pompe disease when she was 15 months old.

Crowley's father John, the President and CEO of Amicus Therapeutics, sat down with Rare Disease Report at the 14th Annual WORLDSymposium in San Diego earlier this month to discuss the impact that one brief mention from the President has had on his family, and on raising awareness for rare diseases as a whole. In this video, he stresses the importance of events like Rare Disease Day to make sure that the United States can continue advancing as it pertains to therapies and treatments for rare disease patients.

Crowley: A year ago, when the President delivered his address to a joint session of congress, it was on the 10th anniversary of World Rare Disease Day. We advocated for a mention of the needs of people living with rare diseases and we were honored that it was more than a mention. It was 2 and a half minutes of that speech. We were honored that our daughter, Megan, was chosen as a representative of not just somebody living with Pompe disease, but somebody living with a rare disease. Megan realized then, as she does today, that she’s a proxy and was especially in that moment for millions and millions of people.

That day allowed us the ability and the days and weeks that followed to be able to raise the awareness of people living with rare diseases, how many there are, how many diseases they live with, what the challenges are, and what the hopes are, and what the dreams are, particularly in the world of making medicines. I hope that contributed to that dialogue in some meaningful way that now maybe there is more research being done, more attention being paid. I think we’ve seen terrific advancements in regulatory science by the FDA, additional resources of the FDA which is critically important, and very importantly, very strong leadership at all levels of FDA, increasingly now focused on advancing medicines as quickly as possible for a range of rare diseases.

That dialogue had already begun long before that speech a year ago, but I think in that moment it really captured the momentum that we have, the wind in our backs, and now we need to make sure that we can advance all these exciting new technologies. So, we have a very exciting year behind us and I think exciting years ahead, as well.


This Rare Disease Day, Rare Disease Report wants to encourage our audience to share helpful and informational content whenever possible in the effort to raise awareness. You can keep up with RDR on Facebook and Twitter, or by subscribing to our e-newsletter.
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