Dad gave up career to find cure for daughter’s rare genetic disorder: ‘Maybe she’ll say ‘daddy’ again’

Casey McPherson, lead singer of Flying Colors, put his music career on hold after his daughter Rose was diagnosed with HNRNPH2-related neurodevelopmental disorder in 2019.

The condition left the once talkative 9-year-old unable to speak, pushing Casey to shift from performing on stage to caring for Rose full time and searching for a cure.

“She screams and giggles and cries and makes noises and sounds, but she doesn’t have any friends,” 46-year-old Casey told PEOPLE.

His daughter has an HNRNPH2-related neurodevelopmental disorder that makes it hard for her to form connections with other children.

He explained that while Rose communicates through sounds and emotions, she struggles to interact or play with others.

Life after diagnosis: A new mission begins

After the diagnosis, Casey made a life-changing decision. He walked away from a Sony record deal to focus entirely on helping his daughter.

In 2023, he founded AlphaRose Therapeutics, a biotech company aiming to create a treatment tailored for Rose.

So far, it has raised over $1 million and is working toward a $5 million goal by June to support clinical trials.

A new treatment has already been developed for HNRNPH2-related neurodevelopmental disorder, and the company is now raising funds and seeking approval to begin trials.

Rose could start treatment within six months if everything goes according to plan.

“This is just the beginning for me. I’m devoting the rest of my life to this,” Casey said.

His journey is featured in Rare, a documentary that follows families searching for cures for children diagnosed with rare and ultra-rare genetic disorder.

The film was recently shown in Washington, D.C., where Casey and others are pushing for clearer rules and more funding for treatments that can help the 30 million Americans living with rare conditions.

The FDA states that more than 7,000 rare diseases each affect fewer than 200,000 people in the U.S., with many having no effective treatment and being life-threatening.

Living with an ultra-rare genetic disorder

Every day matters for Casey and his family as they watch Rose’s health closely.

Despite developing at the level of an 18-month-old, Rose continues to fight through each day.

Her father remains hopeful, praising her determination to survive and engage with the world.

After Rose was born in 2016, she appeared healthy. But she missed key developmental milestones, and concerns grew when she began choking on her food.

Later, she had trouble walking and took years to master it. She never learned to crawl and could only say simple words like “mom,” “dad,” and “outside.”

Her parents visited multiple doctors in search of answers. Their concern deepened when Rose lost the ability to speak and began crying in pain.

This marked the start of what Casey calls a “diagnostic odyssey.”

When Rose was three, they finally received answers—a mutation in the HNRNPH2 gene was causing her symptoms.

She was also diagnosed with autism, which Casey believes is tied to the ultra-rare genetic disorder.

Dr. Jennifer Bain, a child neurologist who has studied the HNRNPH2-related disorder for eight years, said individuals with the mutation often face delays in walking and talking.

Most require full-time care and display signs of autism or related behaviors.

Bain also highlighted the risk of seizures, epilepsy, skill loss, and early death.

“So, it certainly can be a very devastating diagnosis,” she said.

Turning pain into purpose

Rose’s condition has led to multiple medical challenges.

She suffers from unpredictable seizures—one of which stopped her heart for about a minute until her mother performed CPR.

She also struggles with depth perception, remains unpotty-trained, and needs monitoring for oxygen and heartbeat while she sleeps.

These challenges make travel or even short trips difficult, leaving her divorced parents feeling isolated.

Rose’s 11-year-old sister, Weston, often has to take a backseat due to the demands of Rose’s care.

Still, Rose stays connected to the world and attends a private school.

Her father believes she is much smarter than her test results show, explaining she has only lost some abilities.

In 2021, Casey created the To Cure A Rose Foundation. Two years later, he launched AlphaRose Therapeutics, aiming to develop treatments for rare genetic diseases—an area often overlooked by larger drug companies.

Casey remains focused on one goal: helping Rose and children like her, diagnosed with an ultra-rare genetic disorder, gain basic abilities many people take for granted.

“Maybe she’ll make friends. Maybe she’ll sing a song, maybe she’ll say ‘Daddy’ again one day,” Casey said. “Those are my goals. Those are the things that matter to parents.”

Here’s Casey McPherson talking about the story of AlphaRose that started with the diagnosis of his daughter’s ultra-rare genetic disorder: