The Cost of Rare Disease

By Laura Edwards

My family has been fighting Batten disease for many years. Once upon a time, my sister Taylor earned straight As in school and sang the words to all the songs she heard on the radio. She insisted on joining the Girls on the Run program at her school, even though she’d recently lost her vision. She ran two 5K races with her team.

Today Taylor is totally blind, unable to speak, learn or walk, suffers from seizures, and has a feeding tube. Batten disease stole my sister’s childhood. Now it’s stealing her life. Other lives, too, are being changed by Taylor’s illness – those of her family, friends and all who love and care for her.

This is the human cost of rare disease.

There are other costs, too. Multiple trips to the hospital emergency department and several surgeries in a single year cost hundreds of thousands of dollars. The saddest part is that none of these expensive treatments addressed the root cause of Taylor’s illness; they only treated her symptoms.

Taylor is just one example, but the impact of rare disease is unmistakable. My sister deserves better, as do the millions of Americans suffering from a rare disease.

But providing better treatments can help more than just the rare disease community. It can also ease the burden on our healthcare system and the economic burden of rare disease that affects us all.

That’s the message my mom, Taylor’s Tale President Sharon King, delivered to a bipartisan group of North Carolina legislators, industry leaders and patient advocates at a legislative breakfast hosted by Taylor’s Tale in the state capital. She delivered an impassioned speech, much of which I’ve borrowed here, to garner support for new legislation designed to position North Carolina as a leader in rare disease research and development, leading to more and better treatments for people like my sister.

Good things are coming. The response to my mom and her fellow speakers, UNC Gene Therapy Center Director Dr. Jude Samulski and BioCryst CEO Jon Stonehouse, was incredible. My mom’s visionary leadership, coupled with the intellectual firepower of our state’s research universities and biomedical industry, has us positioned to build a bright future for the millions like Taylor.

The sad part of the story is that none of this progress is coming in time for my sister. I know she’s still in there, bottled up somewhere, fighting against the Batten monster. I know that regardless of what tomorrow brings, I’ll never forget how her courage inspired others.

View photos from the breakfast on Facebook

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