Understanding Pompe Disease — Princeton girl Megan Crowley’s story inspired Trump
WASHINGTON D.C — A Jersey Girl with a rare disease was in the spotlight at President Donald Trump's speech to a joint session of Congress on Tuesday.
Megan Crowley, 20, of Princeton was a special guest of Trump's — there to illustrate his call to "slash the restraints" of the "slow and burdensome approval process at the Food and Drug Administration." Crowley suffers from Pompe Disease, which can severely limit body functions.
The sophomore at Notre Dame gave a wave and a smiles as the chamber filled with applause.
"An incredible young woman is with us this evening who should serve as an inspiration to us all," Trump said, telling Crowley's story of being diagnosed with the rare and serious illness when she was 15 months old. She was not initially expected to live past the age of 5.
"On receiving this news, Megan's dad, John, fought with everything he had to save the life of his precious child. He founded a company to look for a cure, and helped develop the drug that saved Megan's life," Trump said, saying her story was "about the unbounded power of a father's love for a daughter."
“I know John Crowley, Megan’s father, and that’s a wonderful story,” Rep. Leonard Lance said after the president's address. “I was proud as a New Jerseyan that the president highlighted Megan Crowley.”
Brendan Frasier played John Crowley in the 2010 movie "Extraordinary Measures" that told the story of how he founded Novazyme Pharmaceuticals and helped develop a drug that kept Megan alive.
What is Pompe Disease?
- Pronounced "pomp-uh," the disease is "an inherited disorder caused by the buildup of a complex sugar called glycogen in the body's cells. The accumulation of glycogen in certain organs and tissues, especially muscles, impairs their ability to function normally," according to the U.S. National Library of Medicine. Researchers have identified three different types of Pompe disease, according to the National Library of Library of Medicine: classic infantile-onset, non-classic infantile-onset, and late-onset, which can vary widely in terms of age of onset and severity.
- According to Crowley's website, highheeledwheels.com researchers have identified up to 300 different mutations.
What are symptoms of Pompe Disease?
- The U.S. National Library of Medicine says Pompe affects about 1 in 40,000 people in the United States. Some of the symptoms include muscle weakness, floppiness, and head lag. Respiratory difficulties are often complicated by lung infection, as described on Crowley's website.
How is Pompe Disease treated?
- There is no cure, according to pompe.com. There are several types of care and treatment can help treat it and a variety of supportive therapies to alleviate and compensate for the effects of the disease. Enzyme replacement therapy (ERT) is available, to replace the deficient acid alpha-glucosidase (GAA) enzyme.
Contact reporter Dan Alexander at Dan.Alexander@townsquaremedia.com.