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The Koujaians

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Harry

Hayley's dad, Harry, fights for her life by pursuing investigational treatments like cyclodextrin.

Gail

Mom Gail is dedicated to fighting alongside her husband to save Hayley's life.

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Alec

Hayley's older brother Alec helps his sister in her fight against NPC.

Hayley

Hayley, the younger of two, began to have seizures in 2009. In Oct 2011, when she was 12 years old, Hayley was officially diagnosed with Niemann-Pick Type C. She fights NPC using cyclodextrin.

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Niemann-Pick Type C

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Symptoms

"...They figured out where the seizures were coming from, and then they operated on her. The seizures continued."

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Cyclodextrin

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Using Cyclodextrin

"The best that we'd hoped for when we started with this journey was stabilizing. She's better than that."

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Early Intervention

"The brain cells that aren't dead, they could be repaired. But anything that is already dead, there's no going back...it's very important to start right away."

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Results

Family

Since starting cyclodextrin treatment, Hayley has regained the ability to feed herself and use the bathroom, among other improvements.

Dr. Elizabeth Kravis

Hayley's ability to walk, turn, and balance has improved as she's been taking cyclodextrin.

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Intrathecal (IT) Cyclodextrin

"Outside of the annoyance of having to...spend a day in clinic every two weeks, what we do is really a pretty overall benign process."

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Pursuing Cyclodextrin

"The family approached me about whether I would be willing to do an IND [Investigational New Drug application] for her to have intrathecal cyclodextrin, because she didn't qualify for the [NIH] trial."

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Recommending Cyclodextrin

"My firm belief right now from what I've seen is that everyone with NPC should in some way have access to cyclodextrin going forward."

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Hope

"Two or three years ago, there was nothing. Now there is."