Rare Disease Report

President Trump Praises 'Miracle' Rare Disease Person Megan Crowley

FEBRUARY 28, 2017
James Radke
President Donald Trump’s First Address to a Joint Session of Congress included a special shout out to 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.
She blogs about her extraordinary life at highheeledwheels.com/
Megan is the daughter of John Crowley, CEO of Amicus Therapeutics. His struggle to find a treatment for Pompe disease was the basis for the Harrison Ford film Extraordinary Measures.

The FDA, Red Tape, and Miracles

While introducing Megan, President Trump noted that Megan was not expected to live past her 5th birthday. The President also said that Megan is a ‘miracle’ and more miracles like her can only occur if the unnecessary bureaucracy that exists in agencies like the FDA are removed.
President Trump said, “Our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan’s life, from reaching those in need. If we slash the restraints, not just at the FDA but across our government, then we will be blessed with more miracles just like Megan.”

About Pompe Disease

Pompe disease is a rare lysosomal disease that may present in childhood (early-onset) or in adulthood (late-onset). In both cases, the disease is due to a deficiency in the enzyme alpha-glucosidase (GAA). Symptoms vary greatly in this population but can be severe and ultimately deadly in the early-onset type.

In an interview with RDR last fall, Priya Kishnani, M.D. of Duke University talks about Pompe disease and the need for more research towards this devastating disease.

Also, below is an interview we did with John Crowley in 2013 about the movie Extraordinary Measures.

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