Casey McPherson was raised in Lake Jackson, Texas. By the age of twenty-three, Casey had lost both his father and brother to suicide. He poured himself into his music to help process his emotions, launching multiple bands with great success. His bands, Alpha Rev and Flying Colors, have toured the world with songs in the Top 10 charts. In addition to a long music career, Casey dedicated over a decade to help others face mental health and prevent suicide. He joined the boards of Mental Health Texas and Austin Child Guidance Center, where he worked on policy change and mental health advocacy.
He got married, and had two daughters: Weston, and Rose.
Before she was born, Rose seemed to be developing normally, perhaps a bit slower than others - but nothing that would cause alarm. She took longer to learn how to crawl, walk, and talk, but she was a happy, growing baby. Her first word was "outside."
Before long though, Rose's parents knew something was wrong.
She missed more and more milestones as she grew up. Rose would fall, but she wouldn't put her hands out to catch herself. In 2019, they received an official diagnosis: her HNRNPH2 gene was mutated, and her body wasn't producing the proteins necessary for her to develop properly.
Rose lost the few words she could say, and continues to lose her ability to communicate to this day. Her motor skills are impaired, and she struggles to feel safe and comfortable in overstimulating situations.
Neurologists told Casey there was nothing they could do. There is no treatment for HNRNPH2.
After Rose's diagnosis, Casey met with hundreds of biotech companies, academic researchers, parents, and foundations. He learned that for Rose's disease, there is a path forward to create a treatment.
HNRNPH2 is a monogenic disorder. There's just one gene that's causing so much pain for these children. With todays technology, we can create a treatment to address a single gene mutation. It's been done before to address similar diseases, like Angelman Syndrome.
To date, around 100 people have been diagnosed with an HNRNPH2 mutation, but some estimates show there could be closer to 10,000 people living with this condition. It's also not a hereditary condition: HNRNPH2 mutations don't appear to be passed down by parents. This is a mutation that will continue to appear for generations to come.
But if we start with Rose - if we create a treatment for her mutation - we can treat so many more Roses:
Every Rose that has been diagnosed. Every Rose that has yet to be. And every Rose that will be born with this condition in the future.
Now, we have a path forward for many other diseases.