Recently, Rose started walking up and down the stairs. I was so excited, because it was a level of autonomy that she hadn't had before. But it comes with a cost, because if she falls even one time it could be catastrophic. I have baby gates for a 6 year old. Her ABA therapist sent me a video of her stepping on a stool to a sink, and everyone was so excited. She was excited. Because she stepped up onto a stool. She can't talk, she can't understand what's going on most of the time, she feels immense pain, she is still in diapers - so those are our wins.
I see her "trying" to do things other people do, pushing herself, but she can only get a small percentage of it. It's like she's got one hand tied behind her back to play baseball. She's surrounded with people to help her, and she's giving everything she's got but, until we remove the rope off her hand, she will always be one handed. And the thought of her losing everything she worked hard for when she reaches puberty is looming. But she pushes herself nonetheless. If she's willing to, how can I not?
We've recently hired Perlara to help us manage the drug development and partnerships on the science end. Her cells are about to be turned into brain cells, her humanized "Rosie" mouse is being created, and we're a couple of months away from our first ASO drug design. We're getting much closer, thanks to you.
Imagine a world where the next child with a rare disease, of the roughly 200m that exist on this globe, has a path forward. Instead of waiting for a pharma company to make a drug in 30 years, and only if it's massively profitable. Imagine a world where this is no longer a dead end but a "procedure" like a surgery or an MRI. A process we can repeat that saves lives.
That's my vision for these children. We have the technology today to make this possible. But the financial models don't work yet for some of these diseases and the process is inefficient. The advancement of this science won't happen without the money. You are making that possible, and it will help change healthcare forever.
While I celebrate the wins for Rose and for our Foundation, We have a steep climb to the top of this mountain, and I keep a sober thought on what the real goal is. We MUST give Rose and all the other Rose's out there a chance at being able to thrive in this lifetime. Rose won't give up, she won't let me give up, and I have a feeling you won't either.
Thank you - because of you, these children are seen, they are heard, and they are being fought for.