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ASHINGTON — A young woman whose father founded a biotechnology company in pursuit of a treatment for her rare disease will be front and center on Tuesday night for President Trump’s address to a joint session of Congress.

Megan Crowley, a college student who was diagnosed with Pompe disease at 15 months old, will be seated alongside first lady Melania Trump during the address. Beforehand, Crowley and her father, John, will meet with Trump in the Oval Office to discuss rare disease treatments.

“Megan and our family will meet privately in the Oval Office with the President to discuss the needs of people living with rare diseases, and especially advancing medicines for treatments and cures for ALL rare diseases,” John F. Crowley wrote in a Facebook post.

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The timing is fortuitous: The visit and Trump’s address come on Rare Disease Day.

Following Megan’s Pompe diagnosis and that of her younger brother Patrick four months later, John Crowley founded Novazyme, which developed protein engineering technologies aimed at treating diseases like Pompe.

Novazyme, which was acquired by Genzyme in 2001, was eventually successful in creating an enzyme replacement therapy for Pompe disease, which Megan and her younger brother are still treated with today.

The Crowleys’ family history has been well-documented over the years, most notably in a Wall Street Journal profile and subsequent book by Geeta Anand, then a film starring Brendan Fraser and Harrison Ford titled “Extraordinary Measures.”

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The pursuit of a functioning Pompe treatment was also the subject of a 2002 Harvard Business School study titled “Father’s Love,” in which Crowley “must choose between a partnership and a buyout to have sufficient funds and support to get the drug to the market.”

Pompe disease, an enzyme deficiency that causes glycogen buildup and leads to muscle and respiratory damage, is estimated to affect 1 in 40,000 people.

Megan is currently a sophomore at the University of Notre Dame. Her father, currently the CEO of Amicus Therapeutics, a biopharma firm specializing in rare and orphan disease treatments, has been repeatedly mentioned as a potential Republican candidate for one of New Jersey’s two Democratic-held Senate seats.

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  • My daughter is 5 and was diagnosed with Pompe at 3 months old! It’s been such a long journey and we are struggling with finances and depression and I was wondering if anyone would be interested in helping our family towards our gofundme campaign? I’ve tried so many other avenues but they’ve all lead to nothing.

  • Very interesting! I recently did an article on how telemedicine can be used to reach remote patients. This story is very interesting as it relates to a few of the trends that we are seeing in the healthcare arena; there will be some major changes to the industry over the next few years.

  • I recently met Katie Meyler after her presentation at Amicus Therapeutics. She is the founder of More Than Me, a nonprofit organization that provides girls with the education, health and social services they need in the Ebola stricken slum of West Point, Liberia. During her caregiver role in the fight against Ebola Katie realized that you cannot wait for medical agencies, governments or aid organizations to come in and “be the hero”. The problem was overwhelming. She needed to “be the hero”. I believe that John Crowley and Megan have received the same revelation and are acting upon it. If only we all would do the same…

  • Has the this drug been use as an experiment for Duchesne’s Muscular Dystrophy. You mentioned muscle damage so that’s why i’m asking. Thank you for time.

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