Rare Disease Report

Trump's Joint Address to Congress to Include Special Rare Disease Guest Megan Crowley

FEBRUARY 27, 2017
James Radke
Tonight, when President Donald Trump speaks at his First Address to a Joint Session of Congress, there will be a special guest sitting with First Lady Melania Trump – Megan Crowley.

Megan is 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.
 
It is hopeful that Megan’s presence tonight will ease many rare disease patients' concerns about how President Trump will change the way chronic, rare, and/or pre-existing conditions are managed and researched. With that said, it is not clear what the current administration’s philosophy is towards the rare disease community other than to note that the healthcare system ‘is a mess’.

Rare Disease Day

Coincidence or not, it should be noted that the presence of Megan Crowley during tonight's Joint Session of Congress could not have been more timely – February 28th is Rare Disease Day.

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, MD talks about Pompe disease and the need for more research for this often devastating disease.

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



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