When his daughter lost her ability to speak, he gave up everything to just hear her call him 'daddy' again

Parents of children with rare genetic conditions often face an uphill battle navigating unknown territory, uncertain prognoses, and limited treatment options. For one Texas father, that journey became a mission that transformed his life. Casey McPherson, a musician and father of two, gave up his professional career to find a cure for his daughter Rose, whose ultra-rare disorder took away her ability to speak. Casey McPherson spoke exclusively to Upworthy about his journey and why he decided to focus on being a dad. "I was scared that I wouldn't have what it takes to save her, and give up a career that held my greatest skill. "What I also didn't know was that my identity was no longer in music, it was in being a father to my girls, and there was nothing I wouldn't do for them, which made me the perfect founder for AlphaRose foundation."

Casey McPherson, known for leading the bands Flying Colors and Alpha Rev, stepped away from the music industry after his daughter, Rose, was diagnosed with HNRNPH2-related neurodevelopmental disorder, a rare condition with just over 150 diagnosed individuals globally, as per a 2024 report from rarechromo.org. The condition, linked to a mutation in the HNRNPH2 gene, has affected Rose’s speech, motor function, and cognitive abilities. She was diagnosed in 2019 at the age of three. "She screams and giggles and cries and makes noises and sounds, but she doesn't have any friends. She doesn't know how to play with anyone," McPherson told People in an interview.

Rose’s health has continued to deteriorate, with symptoms including seizures, impaired mobility, and loss of speech. Dr. Jennifer Bain, a pediatric neurologist who has studied HNRNPH2-related disorders for eight years, explained that most patients with the condition experience severe developmental delays and require lifelong support. "Many of them have autism spectrum disorder or autistic features. So, it certainly can be a very devastating diagnosis," Bain said, adding that individuals with this disorder face an increased risk of seizures, regression, and premature death. In response to the lack of available therapies, McPherson launched a nonprofit, To Cure A Rose Foundation, in 2021. The organization later led to the formation of AlphaRose Therapeutics, a public-benefit corporation focused on developing precision medicine for rare genetic conditions.

It has since raised more than $1 million in funding, with a target of $5 million to initiate clinical trials. "This is just the beginning for me. I'm devoting the rest of my life to this," McPherson said. The company's lead treatment candidate was developed in consultation with genetic researchers and is currently undergoing the process required for clinical trial approval. If successful, Rose could begin receiving treatment within six months. McPherson’s efforts are also featured in the documentary "Rare," recently screened in Washington D.C., which follows families attempting to fund and develop treatments for ultra-rare conditions. McPherson has described his daughter’s daily condition as requiring round-the-clock monitoring. 

Casey McPherson still recalls some of the earliest memories of her, and they are some of his treasured moments. "I remember how small she was when she was born, and bathing her tiny little body in the bathtub, and making sure the water was just right. I remember the first time Rose said 'Dad,' he said in an exclusive interview with Upworthy. before adding how it inspires him fight for her everyday. "I think back about all the hours and days and years I spent holding her hand while she was trying to walk. She never gave up, and so neither did I!"

Her sister, Weston, age 11, has also been affected by the family’s limited mobility and increased care responsibilities. "She's had to take the back seat a lot of times," McPherson said, but his respect and love for Weston have grown infinitely after seeing her watch over Rose.  "Weston is a special young lady. In all reality, she should resent Rose, but instead, Weston cares for her like a motherly older sister. She has learned to find her self-worth in who she is, not the attention she gets. She helps me with Rose and is an active little 11-year-old in our family, exuding grace and wisdom way beyond her years."

McPherson also built a special bond with Rose through music. "No matter how hard the day is, Rose and I have this really special time at 6.45 pm every day, right before she goes to bed. Often, I will get my guitar and sing songs to her. She will sometimes take the harmonica I give her and breathe in it as if we are playing together. Other times, she will giggle and put her hands on my guitar strings to feel the vibrations while I play."   

If there's one thing McPherson was clear about — what it takes to save children like Rose. "I have come to realize that in order to save Rose and millions of children like her, we must fix the system. Which means we must have a sustainable precision medicine path, otherwise foundations and families will always be scrambling to raise millions of dollars to save their little ones," he said. That was what spurred him to start the change. "It's why I started the for-profit PBC, AlphaRose Therapeutics, to show investors and the healthcare community that these small population medicines can be profitable at scale. Now, I get to offer this investment to the entire world by crowdfunding our equity with people that want to change the world for the Roses. If we reach our funding goal of 5 million, we will be able to treat the "Roses", and maybe I get to hear her say "dad" again."

McPherson has called for greater regulatory support to expedite approval of treatments for rare diseases, citing gaps in pharmaceutical investment and the slow pace of clinical research. AlphaRose Therapeutics aims to scale research and treatment models to serve rare patient populations that are frequently overlooked in drug development pipelines. "Rose is working really hard. She's giving everything she's got to stay alive and to exist. So, I should be doing everything I can to give her every possibility of unlocking her freedom," McPherson said. While the road ahead is uncertain, McPherson holds on to small hopes. "Maybe she’ll make friends. Maybe she’ll sing a song, maybe she’ll say 'Daddy' again one day. Those are my goals. Those are the things that matter to parents," he said.