Because One Day Isn't Enough

Rare Disease Day focuses on the millions suffering from 7,000 rare diseases.

PhRMA StaffFebruary 28, 2011

Today is Rare Disease Day, a day meant to raise awareness of the 25 million Americans living with diseases that are often little-known and often, unfortunately, undertreated.

In conjunction with Rare Disease Day, we released our report on orphan medicines in development to treat rare diseases, which found a full 460 medicines currently being studied. That number represents tremendous hope to millions of Americans, though we do recognize that, with up to 7,000 rare diseases around the world, it is just a start.

Here at The Catalyst, I'm extending Rare Disease Day by an extra four days, declaring it Rare Disease Week. Why? Because there are too many stories to tell in just one day, and because the patients deserve it.

And, to be honest, because it's something that I have long cared about.

In elementary school, based on a confluence of circumstances (namely the diagnosis of a friend of my father's at roughly the same time that I saw the movie "The Pride of the Yankees"), I became interested in learning more about amyotrophic lateral sclerosis, also known as Lou Gehrig's disease.

I chose to study the disease for a school project, becoming deeply immersed as I learned about its tragic consequences, and ultimately arranged my own little fundraiser, bringing my family on a four-person, five-mile walkathon.

Together, thanks to coverage from the local newspapers and the resulting donations that flowed in, the Connors family raised several hundred dollars, which I proudly sent to the ALS Association.

Today, working at PhRMA, I know that it can cost more than $1 billion on average to bring a new medicine to the market, and in the field of rare diseases, that price tag can be even higher. So, I imagine my $400 or so (I no longer remember the exact amount) probably didn't get too far.

But working in this sector, which is filled with hope and promise, I also have come to deeply appreciate that every step counts.

Interested in learning more about rare diseases like ALS? Click here to see some videos that we put together about rare diseases and come back throughout the week to see what else has caught our eyes.

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