This is the little girl who used to not be able to sit up without falling over. Who couldn’t push up to stand. Couldn’t take a step. Couldn’t eat yogurt without choking. Couldn’t put two words together and forgot how to wave “bye bye” before she was 1 ½. 

This is the little girl who at 18 months was told she has one of the most aggressive versions of fatal Niemann Pick Type C (NPC), and all of those things were signs neuro degeneration was already happening. Imagine holding your baby, looking in their face, and knowing what is in their future: rapid loss of all skills, choking, seizures, tremors, and after all that, dying. 

This is the little girl who at the time became the youngest patient in the world to get access to an experimental therapy. We went back and forth to Chicago, then to Children’s Hospital Los Angeles for her to get her medicine, every other Friday, without fail. And it’s saved her!

She got stronger again. Her hair grew in. She gained weight again. She started talking more and re-learned how to wave “bye bye.” We thought, wow, this is amazing, we are so lucky to have this medicine. If this is as good as it’s going to get, we are so grateful to have this. 

But then, even more happens, she takes her first steps, and she pushes up to stand off the ground saying, “ta-dah” with a proud smile. She starts ballet class and she learns to spell her name and count to ten. She begins pre-K and then Kindergarten. She is now strong enough to jump, get dressed, climb in and out of her car seat and into her sister’s top bunk on her own. She tells adorably bad knock knock jokes and puts on shows. She makes friends and loves them. All while getting her medicine, every other week. We thought, wow, this is saving her life! We are so grateful to have her, our daughter, who is fighting with every ounce to beat this cruel and relentless disease, and this medicine is all that is standing between her life and NPC taking it away.

But now, this all might get taken away because her medicine is at risk. We think, if it does, will she have another birthday? If she does, what will it look like? Will she be able to be independent? What will the last time we will hear her voice be? When will she need to start being tube fed? Will her body be strong enough to fight off the pneumonias that follow with the loss of swallowing? How will her friends and family remember her? What will life look like as it goes on without her in it? This is why she needs her medicine. It works. 

This is my daughter, Marian, and last week she turned six years old. She needs her medicine. Her NPC friends need their medicine. If they lose their medicine, they will suffer and die. This is urgent. 

Please keep sharing this petition, for my daughter Marian and all NPC patients.

#dontgiveuponnpc