McPherson Family

02.12.2025

When Rosie was little, she learned to say "daddy", "mommy" and "outside", her favorite place to be. She struggled to walk. When she finally got on her feet, she wobbled down the street, pushing her tiny lawnmower, looking up to her dad Casey McPherson with pride, and smiling. But she lost her only words and her walking continued to be unsteady. She began covering her ears in fear and screaming. And she never made it out of diapers or attended a typical school. When she was 3, she was diagnosed with HNRNPH2, a rare condition with a bleak and unknown future. 

Today, Rosie is 9. She spends most of her time at her dad's house, her mom's, and her small school, as loud and busy places normal families would go are too overwhelming for her. She wanders the house in silence, picking up stuffies and jewelry in her sister's room, opening living room drawers and removing the contents, and taking eggs from the fridge and throwing them on the floor. 

Casey has spent the last 4 years fighting for a cutting-edge genetic treatment for Rose in hopes of bringing back even a few words, or giving her the ability to wind a toy on her own or sit on the toilet. But what he's fighting for most is his daughter's ability to connect with the world around her. He watches her stand alone on a playground longing for interaction with the kids who run past her. And he sees how she fills that void with animals who connect without words and do not judge her. And so he has lived in the country where he can surround Rosie with pigs, goats, chickens and dogs. And while he fights for her, Rosie can find that special connection. 

02.13.2025

As a single dad of two girls with completely different needs, Casey struggles to take care of Rose while still giving his older daughter, Weston, the attention she deserves. Rose needs to be dressed, bathed, given water and food, given her medicines, and have her diaper changed. Weston wants to tell her dad about school and her friends, do art projects together and have him listen to her favorite songs. She wants to go to the movies, play soccer or take a trip. But Wes' needs always seem to come second as Rose is fully dependent on her dad.

After a long day of work and school, Casey sits at the piano with Rose, allowing music to so beautifully connect them. But his joy is balanced with the pain of knowing how alone Weston feels, always aware of where she is and how she has had to entertain herself since Rosie was diagnosed.

02.18.2025

For Casey, each day can feel like survival. Taking care of Rose's basic needs and constantly cleaning up her trail of toys, books and food thrown around the house can push him to the edge. But at the end of each day, he has their nighttime routine to look forward to, a little window when Rose calms and connects with him.

After dinner, Casey holds Rose's hand and they walk slowly up the stairs to the bath. She plays with the bubbles and holds her cup under the dripping faucet, smiling up at him. Then he picks her up, wraps her in a towel and swings her onto the bed, bouncing onto the pillows, her laughter filling the bedroom. He grabs his guitar and sings her a song, "Rosie, Rosie, what are you thinking…”. She looks into his eyes and bounces on her knees in excitement. He pauses, letting her play the next verse on her harmonica, and they go back and forth until she lays her head on his chest and sticks her thumb in her mouth.

This little moment of connection at the end of each day, the music they compose together, makes all of the hard stuff worth it.

02.20.2025

Without a treatment, what will Rosie’s life look like? As she ages, will she continue to depend on the cartoons and movies she’s watched as a toddler for connection with the world around her? Will she be able to feed herself and know when to go to the bathroom? Will she be able to live independently or fall in love? Will she survive to adulthood? And if she does, who will take care of her when her parents are gone?

These are the uncertainties that drove Casey to leave his music career, the one he worked on day and night for 25 years, to fight for his daughter. As a lead singer and song-writer who pushed through life under the poverty line to form a successful band, sign major record label deals and watch his songs become top hits, he now finds himself pulling from that same grit and determination to learn drug development, raise the millions needed and put the scientists and doctors together to create a new business model for rare disease treatments that relieves the burden of hope that falls so heavily on families like his.

Casey dreams of a brighter future for Rose where real relationships take the place of cartoon faces on screens, and a model of access that offers the same future to other Roses across countless diseases like hers.