My Rare Disease Day Dream

rdd-logo-2It is fitting for Rare Disease Day to mark the end of Black History Month this year, for in reflecting on the current state of our nation, as the civil rights activists did decades ago, the Rare Disease community is faced with a fundamentally synonymous problem: exclusion from the narrative. In the 1960s the famous Baptist minister from Montgomery, Alabama, Dr. Martin Luther King Jr. counseled his fellow black Americans in fighting for inclusion by resisting segregation, racism, and injustice. His goal was a specific, intentional form of resistance: “strong, steady, loving, believing,” and most memorably, nonviolent. The people he led on marches and sit-ins did not follow blindly, though. Dr. King was often asked, “Isn’t negotiation a better path?” To this he offered this simple, yet powerful response: “Indeed, this is the very purpose of direct action.” He recognized these efforts as the only way to “dramatize the issue” to the point it could no longer be ignored.  Nonviolent direct action sought to compel a community, that consistently refused to negotiate, to confront the issue with representatives from all parties–major or minor, oppressive or oppressed, powerful or powerless. 

Although they wouldn’t necessarily identify themselves as such, I had the humbling experience of working alongside nonviolent direct action seekers this summer at Amicus Therapeutics, a biopharmaceutical firm dedicated to the development of “advanced therapies to treat a range of devastating rare and orphan diseases.” The company’s efforts are centered around their mission to discover treatments for the unrepresented minority of healthcare. What makes their method of obtaining these ends unique and particularly noble is their commitment to keeping the patients center-stage. Amicus forges forward on promising research and drug development, all the while staying ever-mindful that the sufferers of rare diseases are often left to flounder on the periphery of mainstream medicine today. Thanks to Amicus and organizations like the Global Genes Project, people with rare diseases are no longer excluded from the health care narrative. They are invited to the table to negotiate. 

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A “rare” friend and fellow “Domer,” Megan Crowley, uses her strong (and often humorous) voice to educate others on her life with Pompe Disease. To read about her amazing story, check out her blog: http://www.highheeledwheels.com
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The valiant efforts of Megan and the Crowley family earned them a private meeting with President Trump in Washington, DC this evening. Regardless of our political beliefs, we should appreciate this as a well-deserved opportunity for them to advocate on behalf of the community they nobly represent. (www.statnews.com/2017/02/28/trump-address-pompe-rare-disease/)

Just as Dr. King’s “dream” inspired a better existence for his people, my dream is that one day the sufferers of rare diseases will enjoy this privilege, too. For today, though, my hope is that anyone who reads this takes a moment to recognize their life is just that: a privilege, something to celebrate and cherish. Our country was once moved by civil rights activists giving their lives for a cause. Today, let us honor the people with rare diseases simply living their lives, an act that is nothing short of heroic. 

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