World Rare Disease Day 2013

By Laura Edwards

This morning, about 80 people joined Taylor’s Tale as we recognized World Rare Disease Day and made an exciting announcement about the fight against Batten disease.

I shared our family’s journey and introduced our featured guest. Following is a modified version of my comments.

My “little” sister is 14. We’re the same height.

But I still remember the day she came home from the hospital. I raced my then-best friend and now-husband up the stairs to peer over her crib (he got there first).

I remember how my sister learned to read as a toddler.

I remember 5-year-old Taylor. She seemed perfect. Beautiful. Intelligent. Healthy. Spunky. She kept our brother on his toes. The world – and Stephen – were hers to conquer.

But 6-year-old Taylor couldn’t handle first grade math. Addition and subtraction left her in tears.

Seven-year-old Taylor couldn’t find her way in dim places.

Just three weeks before my sister’s eighth birthday, a monster called Batten disease burst into our world and shattered it into a thousand little pieces.

Batten disease is a rare, inherited disorder that affects mainly children. To get it, kids have to inherit a “bad copy” of the gene from each of their parents. I got one good copy and one bad copy, so I’m a carrier; that means that I’m healthy but could pass the gene on to my children. My brother is a carrier, too.

Taylor got two bad copies.

With one roll of the genetic dice, our little sister got a fatal disease.

Fourteen-year-old Taylor lives in a world that’s always dark. She can’t learn like other kids. She has seizures. She loves to sing, but soon, Batten disease will steal her speech. Even now, she only has a few words. I can ask my sister if she had a good day at school, but we can’t talk about it.

Taylor ran two 5K races with the help of a sighted running buddy through the Girls on the Run program at her school. But that was four long years ago. Soon, my sister will be in a wheelchair.

Batten disease steals the lives of children.

It upsets the natural order of things.

Children aren’t supposed to have their dreams snatched away from them by a monster like Batten disease.

They aren’t supposed to die.

Taylor was diagnosed with infantile Batten disease on July 24, 2006.

I still remember the long moment we all shared in the floor of my parents’ dark bedroom; wrapped in each others’ arms; soaked in each others’ tears.

The doctor who delivered that news told my parents to take her home and make happy memories.

We don’t have anything against happy memories. But while Batten disease is in our DNA, going down without a fight is NOT.

Every day with Batten disease is a hard day. Still, in the near-seven years since the day our lives changed forever, we followed the doctor’s orders. We made our fair share of happy memories.

But we made time for fighting, too.

With the help of good friends, we founded a non-profit organization. We raised nearly half a million dollars for potentially life-saving research and promoted awareness of Batten disease. We advocated for increased support for the rare disease community.

We partnered with other groups that share our mission; American organizations such as the Batten Disease Support & Research Association and EveryLife Foundation for Rare Diseases, and international organizations such as the Global Genes Project.

And along the way, we met MANY families like ours.

One in 10 Americans suffers from a rare disease.

Chances are, you know someone fighting his or her own battle.

To borrow words from my mom, diseases like Batten are rare…but hope should not be.

That’s why I’m pleased to share reason for new hope with you today.

On behalf of Taylor’s Tale, I’m excited to announce that we’ve joined an international coalition of organizations founded by families like ours – families that refused to go down without a fight.

Together, we’ll fund gene therapy for two forms of Batten disease. This revolutionary work will take place right here in our home state at the University of North Carolina at Chapel Hill. The goal of this study is to pave the way for a human clinical trial.

I’ve watched this disease try to rob Taylor of EVERYTHING. I’ve prepared myself for the reality that I will likely outlive my little sister.

Bottom line – I HATE Batten disease. But in seven difficult, painful years, I’ve witnessed great progress. I’ve NEVER stopped believing.

And I believe in the ability of THIS project at UNC to help save the lives of children like Taylor.

Its leader is an expert in AAV vector design and gene therapy. He graduated from Auburn University and earned a PhD from Vanderbilt University. He completed a postdoctoral fellowship at UNC, specializing in central nervous system gene therapy.  His work focuses on the development of treatments for neurological diseases. He’s been published in multiple journals and mentioned in high-profile publications and blogs. His work is chronicled on the pages of a 2012 book that explores gene therapy as the next frontier through the stories of real patients and families.  The doors of his lab are decorated with photos of the children for whom he and his colleagues go to work every day.

The leader of the Global Gene Transfer for Batten Disease project is Steven Gray, PhD.

To learn more about this exciting initiative, click here.

group at Rare Disease Day event


mir*a*cle

By Laura Edwards

mir*a*clenoun. 1: an extraordinary event manifesting divine intervention in human affairs / 2: an extremely outstanding or unusual event, thing, or accomplishment

On her way out the door following our ACC tournament fundraiser late this afternoon, a woman I’d never met walked up to me, squeezed my arm, looked right into my eyes, and asked the question that forever looms in our anxious hearts: “Is she going to be okay?”

“She” is my sister, Taylor, who at that moment sat less than ten feet behind me at our family’s table and yet was quite clearly wrapped up in her own private, dark world far, far away. Without looking away from the woman’s searching eyes, I offered only the following: “We still believe in miracles.”

I dodged the woman’s question, I know – but the prospect of answering directly quite honestly scares me these days. Each time the sun rises and falls, marking the end of another day without a cure for infantile Batten disease, Taylor’s survival more clearly defies all logic.

When scientists finally unlock the key to this evil disease, I will not call it a miracle. I will call it great science. And I know it will happen. The question is when.

If my sister should beat this disease – that will be a miracle, and when it happens, I will fall to my knees, look up at the sky, and thank God, because no matter what marvels modern medicine can conjure to make her road more comfortable, only He can ultimately lead her out of the darkness.

I’m still waiting for that miracle. But I’ve witnessed other miracles along the way.

Last night, Taylor attended her school dance. When my parents met John and me in the school parking lot to deliver her to us, my mom had tears in her eyes, because in our world, every ‘normal’ experience is emotionally charged. We smile and laugh on the outside, but on the inside, we wonder, ‘Will she be able to handle it?’ and ‘Will this be her last one?’

As we walked down the short hallway to the cafeteria, where the dance had already started, I worried that the kids would ignore Taylor. I silently thanked God for my husband, knowing he would take Taylor’s hand and lead her onto the dance floor if no one else would.

But my fears were unnecessary.

True story: three boys danced with Taylor last night. As I watched from my wallflower spot, an uncanny warmth spread from my head to my toes. And in those moments, I knew I was witnessing a miracle in its purest form – an extraordinary event manifesting divine intervention in human affairs. Because those were angels twirling my sister around the room.


2010

By Laura Edwards

A lot has happened in 2009. I think back now to one year ago today, to how much the prospect of 2009 scared me. Much of what I feared has come true. My sister’s disease has continued to march on; the signs of progression are more evident with each passing day that we don’t have a cure. Much of what I looked forward to, though, also came true. We had good days. We funded research. Research moved forward. After every hard rain, the clouds parted, and the light of the sun washed over us.

2010 scares me more than 2009 did in many ways, but that doesn’t mean that I’ve ever stopped believing. I have no doubt that we will have more bad days next year. Despite that fact, I have no doubt that great things will also happen. Incredible progress will be made. Our faith in miracles will be renewed again and again. And one day – in 2010 or in the years soon to follow, I will use this very space to tell you that we have won.
Thank you for supporting Taylor’s Tale in 2009. Stick with us awhile longer – we have more of the story to tell. Here’s to a promising 2010. Happy New Year!

Light Tomorrow

By Laura Edwards

One of my favorite blogs is written by Molly Barker. Molly is the founder of Girls on the Run International, an organization that has brought much happiness to my sister, Taylor, and helped inspire those around her. I always love Molly’s posts, but her most recent entry wrapped its arms around my heart and wouldn’t let go.
In Her Beating Heart, Molly tells the story of a little girl named Maddie, a girl who, like Taylor, overcame a severe physical handicap to cross the finish line of a Girls on the Run 5K. A girl who, like my sister, refused to be resigned to watching the race – or life – from the sidelines.
I often write about running on this blog. Running plays a powerful role in my life; the act of lacing up my shoes, the rhythmic slap, slap of the soles on firm ground, and the gulps of fresh air that pour into my lungs make me feel like I’m floating through clouds and fill my mind with ideas that I struggle to invoke when I’m sitting still. It’s on my runs that the power of ‘believe’ overtakes my fear of the darkness that threatens my sister’s tomorrows. My heart races not from overexertion but from the thrill of knowing miracles are within our reach. It’s then that I renew my vow to summon within myself even a sliver of the courage that lives within Taylor – to find a way to light her tomorrows.
The fighting spirit that shines brightly within Taylor has kept her off the sidelines until now. By supporting Taylor’s Tale, you can help ensure that children with Batten disease finish the race. Click here to make a gift and keep alive the research that must continue to give them a chance.
In honor of Maddie and Taylor and every child with a dream, I ask – what action will you take today to light their tomorrows?

Grant Summaries are Online

By Laura Edwards

Summaries of research grants awarded by Taylor’s Tale and our funding partners (Hayden’s Batten Disease Foundation Inc. and the North American and Australian chapters of BDSRA) last month are now posted on our website. Click here to learn more!