THE JOURNEY TO CURE ROSE
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Is this the first you're hearing about To Cure a Rose Foundation?
Watch the video below, and scroll down to read our story.
OUR STORY
I'm Casey McPherson.
Before becoming a father, music was my life.
Songwriting and performing around the world was my way of surviving after losing both my dad and brother at a young age.
But when I was told I might also lose my daughter Rose, the music stopped.
Rose is beautiful, kind, and determined. She’s unique. She has no words, but she tells me what she’s thinking through her eyes.
When she’s happy, her laughter lights up the room. When she’s sad, her screams pierce the air.
Sometimes, she looks at things I don’t see and cries when life is calm.
Rose has a devastating rare genetic disease, HNRNPH2.
Children with this disorder struggle to connect to their parents and siblings, to play with kids and attend school, to tolerate sounds and sensations of everyday life.
Some children have seizures. Some die young. Their future is unknown.
Rose has lost her ability to talk.
Walking is difficult, and she struggles with stairs.
She screams in pain and confusion, and can't tell me what hurts...
Rose can no longer say “daddy”
She continues to retreat into a world I’m not part of.
I fear her life will only get more painful, and could end too soon.
BUT TODAY THERE IS HOPE
Soon after Rose's diagnosis, I met with other rare disease parents, researchers, biotech leaders, and countless others, and realized that this can be done. I can save my daughter.
To Cure a Rose Foundation was born.
Yesterday, this would not have been possible, but today, recent genetic treatments have shown that diseases similar to Rose’s can be stopped, even improved.
These are some of those parents that have done this before, and they're helping us.
MILA'S MIRACLE FOUNDATION
Mila's mother, Julia Vitarello created an ASO for her daughter's disease, CLN7 in 12 months. She extended her daughter's life for years with this treatment and brought the science forward for the entire rare disease community. Julia serves on the board of To Cure a Rose Foundation.
SLC6A1 CONNECT
Amber Freed, Maxwell's mother, is working on a gene therapy that is showing promise, and has discovered a small molecule drug that shows amazing results. It is set to begin clinical trials soon.
FAST (ANGELMANS)
Allyson Berent, the mother of 3 children, one of which has Angelman's Syndrome. Through her FAST organization, GENETX startup, and partnership with Ultragenyx the first child was treated with their new ASO therapeutic in 2020. Some of these children regained speech.
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We have our science team.
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We've developed Rosie cell lines and mouse models
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We are close to designing and testing the first iteration of the treatment.
We could have a genetic treatment ready for Rose by next year.
The burden falls on us to raise the money to continue this program in time for her.
Rosie will turn seven this year. If we want to help her, we need to develop a treatment before she hits puberty.
Our window of opportunity is closing.
WE HAVE ALREADY COME SO FAR
OUR PIPELINE
WE CAN'T DO IT ALONE
We are on track to reach proof of concept by early 2023 - but getting there means we need to raise $1.5 million this year, and $5 million in total over the next few years.
We need YOUR help to fund this ground-breaking work toward a treatment.
Without it, there is no hope for a brighter future for her, or any of the other children like her.
SO THAT'S THE BEGINNING OF OUR STORY
If you'd like more details, take a breather and check out what we've got going on with some of the links below.
Meet our team, learn more about HNRNPH2, our programs, and rare disease as a whole, or check out where our next event is so you can come support us in person.
Are you still here?
Good!
Because The Story Doesn't End There.
Just Curing Rose is Not Enough...
THE SYSTEM NEEDS TO CHANGE
Every day, children are diagnosed with rare diseases with no cures. Parents just like me start foundations, and raise millions of dollars in hopes of curing their children.
We are all following the same path, we all share the same goal, but there is no roadmap: we're all starting from the bottom, with no direction and no clue where to begin.
So much time, money, and energy is wasted by this process, and parent's don't get the opportunity to focus on what matters:
Just being there for their kids.
SO THAT'S WHY WE'RE HERE
I want to be the last family foundation for rare disease.
At To Cure a Rose Foundation, we want to carve out a pathway to make developing these treatments more efficient and affordable.
We want the next child diagnosed with a rare disease to have a path forward and fighting chance, whether there is a current treatment or not. Right now, we are working with Dell Children's, Simons Foundation, Jackson Labs, Perlara, and many others to make this vision come true.
150 million children suffer from this, and we can't do it alone.
We absolutely need you in this fight.
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