Rx for a Rare Disease: Create a Mob

Ashley and Donna Appell, leaders in the Hermansky-Pudlak Syndrome Network

Ashley and Donna Appell

Here’s the problem with rare diseases: they’re rare. Not many people have them.

Unfortunately, the average cost for developing a new drug to treat any disease currently runs between $350 million and $5 billion. If only a few thousand people have a particular disease, the numbers just don’t work out. Who has the time or money to research such rare diseases?

As recounted in a recent segment on Marketplace, that was the problem faced by Donna Appell. Her infant daughter Ashley had a rare congenital disease called Hermansky-Pudlak syndrome (HPS), which would probably kill her within thirty years.

What’s a mother to do?

Here it is in Donna Appell’s own words: “Really who’s gonna care about one person? I just had this feeling like we needed to create a mob.”

Sometimes mobs are a good thing!

The good mob

So she set to work forming an organization that would become the Hermansky-Pudlak Syndrome Network.

What can one person do?

Alone, not much.

But by forming a “mob” with other families and friends of people suffering from the same rare disease, Donna Appell was able to accomplish much more. The organization she founded twenty years ago, when her daughter was still young, put HPS on the map. It raised awareness of the disease, provided a community for those affected by it, and created a vehicle to work toward more understanding of HPS and to search for solutions.

Here is a trailer RARE, for a documentary about Donna Appell’s impressive efforts to bring people together in a quest for a cure for HPS:

You can find out more about the film at its official website.

The HPS movement’s slogan is “Dare to be Rare.”

Through its efforts, research into Hermansky-Pudlak syndrome has begun, and some answers are beginning to emerge.

Use a bigger hammer

You see, a mob is like an individual, but bigger.

When a whole group of people gets together for a common cause, it can accomplish much bigger things than one individual can accomplish alone. Every new person in the group adds to the power of the whole.

Each person also brings a unique set of skills and experiences. Different people take on different roles and different tasks in the group. This makes possible a division of labor in which each person can do what he or she is best at.

The result is a whole body of people united to accomplish greater things.

Is there some issue you’re concerned about that nobody seems to be paying any attention to?

Maybe it’s time to create a mob!


Lee Woofenden is an ordained minister, writer, editor, translator, and teacher. He enjoys taking spiritual insights from the Bible and the writings of Emanuel Swedenborg and putting them into plain English as guides for everyday life.

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Lee & Annette Woofenden

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