Musician Casey McPherson, rock frontman of the band Flying Colors, has dedicated himself to finding a cure for his daughter, Rose, who was diagnosed with an ultra-rare genetic disorder. Despite losing her ability to speak and struggling to interact with others, Casey is determined to help his daughter regain her health and independence.
After turning down a record deal to focus on his daughter’s condition, Casey founded AlphaRose Therapeutics in 2023, raising funds to develop a precision medicine treatment for Rose. With the support of experts in the field, the company is preparing for a clinical trial that could potentially improve Rose’s quality of life in the upcoming months.
Casey’s relentless pursuit for a cure is highlighted in the documentary “Rare,” shedding light on the challenges faced by families with rare disorders. He aims to raise awareness and advocate for better treatment options for the millions of individuals affected by rare diseases in the United States.
As Rose continues to work hard to overcome her developmental setbacks, Casey remains hopeful and dedicated to providing her with every opportunity to thrive. Despite the obstacles, he is committed to ensuring that Rose has the chance to unlock her full potential and lead a fulfilling life.
When Rose lost her ability to speak, everything changed. “She would start screaming and she looked like she was in pain,” recalls her father. This marked the beginning of a long journey for the McPherson family as they navigated a series of medical challenges to find a diagnosis and treatment for Rose’s rare genetic condition.
At the age of three, Rose was finally diagnosed with a disorder resulting from a gene mutation known as HNRNPH2. Additionally, she has autism, which her father attributes to the genetic disorder. Dr. Jennifer Bain, a child neurologist at NewYork-Presbyterian/Columbia University Irving Medical Center, has been researching HNRNPH2-related neurodevelopmental disorder for many years. She explains that individuals with mutations in this gene often face developmental delays affecting their motor and communication skills, requiring lifelong support.
Bain further notes that individuals with this disorder may also experience additional challenges such as autism spectrum disorder, early death risks, seizures, and epilepsy. The diagnosis can be devastating for families like the McPhersons, who must cope with the daily struggles of caring for Rose, including her seizures, mobility issues, and the need for constant monitoring.
Despite the hardships, Rose continues to persevere, attending a private school and exhibiting a strong will to engage with the world around her. Inspired by his daughter’s resilience, McPherson founded the To Cure A Rose Foundation in 2021, followed by AlphaRose Therapeutics in 2023, with a mission to develop treatments for rare genetic diseases.
The ultimate goal for McPherson is to see children like Rose thrive and achieve milestones that seemed out of reach. With hope for better treatments in the future, he envisions a day when Rose will walk, swim, make friends, and perhaps even speak again. It is the unwavering love and dedication of parents like McPherson that drive them to pursue a brighter future for their children.
