The Latest Efforts from “Pompe Hero” John Crowley


Those of you fluent in “Pompe 101” are familiar with the efforts of John Crowley. His family is was the inspiration behind the movie Extraordinary Measures. John Crowley’s dedication to getting a treatment for his children with Pompe helped paved the way for all of us to receive the life saving enzyme replacement therapies, Myozyme and Lumizyme. John recently posted the following:

Taking the Pompe Fight to DC!

“RARE DISEASE CAUCUS IN HOUSE….Last week, Congressman Fred Upton (R-MI) and Congressman Joseph Crowley (D-NY) announced the formal formation of the House Rare and Neglected Diseases Caucus. Please see the attached announcement from National Organization for Rare Disorders (NORD).


This is a HUGE step forward in laying the groundwork for a series of hearings, initiatives and ultimately laws that will fundamentally advance the development of medicines for the tens of millions of people worldwide who suffer from rare diseases.

Please contact your Congressman and ask that he/she sign on to the Rare and Neglected Disease caucus in the House, per the “Dear Colleague” letter that Reps. Upton and Crowley sent out to all members of the House last week.

As co-chairs of the Caucus, Crowley and Upton will focus on:

  • Bringing Congressional attention to the 6,800 known rare diseases that currently have no approved therapies;
  • Ensuring sufficient funding for research and orphan product development;
  • Exploring ways to incentivize companies to create new drugs, biologics and humanitarian use devices;
  • Providing an opportunity for Members of Congress, families and advocacy groups to exchange ideas and policy concerns.

Both Rep. Crowley and Rep. Upton are looking to you to encourage your Representative to become a member of this important Caucus. Call the U.S. Capitol Switchboard at (202) 224-3121 today and ask to be connected to your Representative’s office.

If you’re not sure who your Representative is, go to and enter your zip code to find out the name.

Also, FYI, I have been asked to testify this Wednesday before the Senate HELP (Health, Labor, Education and Pensions) Committee on the current state of rare disease research and development. Working late tonight on the testimony draft!!!”

Thank you John for all you do!

UPDATE: To view John’s testimony CLICK HERE, he speaks about 117 minutes into the video.


    1. I would love to do that someday! Take the fight to Washington on an even bigger stage than I did in April! Scary like Tower of Terror scary right? 😉

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