IMAGE: CNS photo/Jonathan Ernst, Reuters
By Dennis Sadowski
WASHINGTON
(CNS) — Megan Crowley,
a University of Notre Dame student born with a rare disease, was recognized by
President Donald Trump during his address to a joint session of Congress.
The
president Feb. 28 acknowledged Megan Crowley, 20, who has Pompe disease, for her strength and character while living with
the inherited disorder since being diagnosed at 15 months of age.
Trump pointed
to Megan Crowley, who was seated in a wheelchair in the House of Representatives
gallery and received two standing ovations, on Rare Disease Day. Traditionally marked on the last
day of February, the day first was designated in 2008 by the European Organization for Rare Diseases.
Megan Crowley
was not expected to live past 5, Trump said, but her father, John Crowley, founded a
pharmaceutical company to develop the enzyme replacement treatment she needs to
cope with symptoms of the disease.
“Megan’s
story is about the unbounded power of a father’s love for a daughter,”
Trump said.
Parents
John and Aileen Crowley attended the address. Megan Crowley’s younger brother,
Patrick, 18, also has Pompe disease. Another brother, John, 22, attends Holy
Cross College in South Bend, Indiana. The college is a neighboring campus to Notre Dame.
Megan
Crowley’s father wrote about the family experience in Congress and the White
House on Facebook Feb. 28.
“I cannot
tell you what it means as parents for me and Aileen to watch as the president
of the United States and all of our nation’s leaders give your daughter two
standing ovations for her strength, courage and character. I felt that my heart
was literally bursting with pride. Just the most amazing young lady. And now
the world knows our Megan. … I knew someday it would,” his post said.
The
president cited the family’s situation as a reason to improve the “slow
and burdensome approval process” at the Food and Drug Administration that
he said has kept advances in treatments from reaching people in need.
“If
we slash the restrains, not just at the FDA, but across our government, then we
will be blessed with far more miracles like Megan,” Trump said.
Pompe
disease is a rare and inherited disorder, occurring in 1 of about every 40,000
births. Someone with the disease experiences progressive weakness in skeletal muscles
and the heart and hampered breathing, and usually dies at a young age. The
disease prevents the body from producing an enzyme that breaks down stored
sugar, called glycogen, into glucose for use by the body. As a result, excessive
amounts of glycogen accumulate in body tissues, leading to major damage.
John
Crowley founded Novazyme
Pharmaceuticals in 1999 to develop protein engineering technologies to
treat diseases such as Pompe. Genzyme
Corp. acquired the company in 2001 and Novazyme eventually developed the
enzyme replacement therapy the young Crowleys continue to use.
Today, John
Crowley is CEO of Amicus Therapeutics, a Cranbury,
New Jersey, biotechnology firm with more than 250 employees. The company
specializes in therapies for rare and orphan diseases.
A 2010 drama,
“Extraordinary Measures,” centers on the efforts of the Crowleys to
find a researcher who might develop a cure for their two children’s rare
genetic disorder.
Megan
Crowley has a blog, High
Heeled Wheels, where she writes about life and her experiences living with the
disease. She also serves as president of Notre Dame’s Make-A-Wish Foundation chapter.
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Follow Sadowski on Twitter: @DennisSadowski.
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